Super Bowl XLIV RAFFLE by cometjunkie47


									                                                                                    As a way to raise money year-round Jeanne
    Super Bowl XLIV                                                                 Muchnick’s friend, Robin Daas and her
                                                                                    company, Causology have created bracelets
        RAFFLE                                                                      with information inside the packaging about
                                                                                    GSD. They can be customized…and are a
    Winning prize package                                                           great thing to have kids go around and sell.
                                                                                    The bracelets are often made by teenage
          includes:                                                                 girls who get community service hours for                                                              The Newsletter for Friends of
•      Two tickets to Super Bowl XLIV, Miami, Florida                               their efforts. It’s a great and easy way to
                                                                                                                                                                         The Children’s Fund for Glycogen Storage Disease Research, Inc.
                                                                                    fundraise. For more information, contact
•      5 days/4 nights accommodations at the Miami                        
       Airport Hotel                                                                                                                                                     NOVEMBER 2009                        GSD NEWSLETTER                            VOLUME 7
•      Two playing spots in the 25th Annual Super Bowl                                                                                                                      This has been a challenging year for non-profit                    are proud to say that after almost 8 years, we are
       XLIV NFL Charities Celebrity Golf Classic
                                                          To obtain a copy of our documentary DVD                                            Contact Us                     organizations and The Children’s Fund is no
                                                                                                                                                                                                                                              still run entirely by volunteers allowing for nearly
                                                          film Life By the Clock for fundraising and/or                              917 Bethany Mountain Road               exception. Every single donation counts, as GSD                   100 percent of every dollar raised, to go directly
•      Two playing spots in Fifth Annual Super Bowl NFL   educational purposes, please contact us at                                    Cheshire, CT 06410                  research continues to progress in the right direction.            into GSD research. To date, we have raised over $4
                                                                                                                                                                                                                                              million dollars and almost all of this has gone into
       Charities Celebrity Bowling Classic       or call (203) 272-CURE.                                    (203) 272-CURE (2873)                 We thank you for making contributions which allow
                                                                                                                                                                                                                                              GSD1 related research. Please visit our website for
                                                                                                                                                                            our foundation to support the research projects that
             Drawing to be held:                                                                                                             WEBSITE                        our scientific advisory board deems worthy.                        a complete listing of research we have supported
          Sunday, December 13, 2009 at 5 PM                                                                                                        We are so grateful for the support we continue                    since the start of our foundation, 7 years ago.
                                                                                                                                                                            to receive from GSD families who lead the way in                  We remain committed to raise funds to fulfill our
                   Ticket Price: $100.                                                    For information on how to obtain                    E-MAIL                        our fundraising efforts, and thankful for those                   ‘Hopes and Dreams’ and our Mission Statement. As
        A Maximum of 1.000 tickets will be sold.                                           "green" GSD reusable shopping                          families who have recently joined our cause. We                   always, we will not stop until we find a cure.
         For more information, please contact                                             bags, please contact Lisa Hodes                                                                                                         Research We Have Supported in 2009
            Teri Reed at (203) 972-9199 or                                                    at or                  MISSION STATEMENT:                                                                       A multicenter, double-blind crossover study of the
                                                                                                                                                                              Dr. David Weinstein, University of Florida
                                                                      call (954) 263-1631                 We are committed to                          Dr. Priya Kishnani, Duke University
                                                                                                                                                                                                                                  dosing and efficiency of modified resistant                $54,541
                                                                                                                                                                                                                                      cornstarch in patients with GSD1a
                                                                                                                                   funding research so that                     Dr. Dwight Koeberl / Dr. Carlos Pinto           Pre-clinical development of a highly efficacious
                                                                                                                                                                                          Duke University                                  AAV vector in GSD1a dogs
                                                                                                                                   children born with GSD1
                                                                                                                                                                               Dr. Thomas Conlon/ Dr. Catherine Mah
                                                                                                                                    will benefit from early                             University of Florida
                                                                                                                                                                                                                                           Gene Therapy for GSD1a                         $100,000

                                                                                                                                   detection, treatment and                                                Research Projects Pending Approval by our Scientific Advisory Board:
                                                                                                                                       an eventual cure.                     Dr. Thomas Conlon, Dr. David Weinstein and
                                                                                                                                                                                                                                           Gene Therapy for GSD1a                   $570,553 over two years
                                                                                                                                                                                  Dr. Barry Byrne, University of Florida
                                                                                                                                                                                                                                  Pre-Clinical Development of AAV Vectors for
                                                                                                                                        Board of Directors                           Dwight Koeberl, Duke Univ.
                                                                                                                                                                                                                                            Gene Therapy in GSD1a
                                                                                                                                   Harriet Bonelli      Michael Julius
                                                                                                                                   David Feldman          Fran Reed
                                                                                                                                   Wendy Feldman          Teri Reed
                                                                                                                                     Lisa Hodes         Valerie Rosen
                                                                                                                                     FUNDRAISING COMMITTEE CHAIR
                                                                                                                                                Fran Reed
                                                                                                                                       SCIENTIFIC ADVISORY BOARD
                                                                                                                                           Andrew Arnold, MD
                                                                                                                                          UCONN Health Center
                                                                       RETURN SERVICE REQUESTED
                                                                                                                                         Hayden Bosworth, PhD
                                                                                                                                     Veterans Affairs Medical Center
                                                                                                                                           YT Chen, MD, PhD
                                                                  917 Bethany Mountain Road Cheshire, CT 06410                       Duke University Medical Center
                                                                                                                                        Academia Sinica, Taiwan
    Permit No. 29
    Cheshire, CT                                                                                                                          David Feldman, DMD
                                                                                                                                    Pramod K. Mistry, MD, PhD, FRCP
                                                                                                                                      Yale Univ. School of Medicine
                                                                                                                                        Mary Beth Schmidt, PhD                                      “...because every child deserves to be healthy.”
            T he Children’s Fu n d                                                                                                                                        A Huge Canadian Success
The research funded by The Children’s Fund for GSD Research is improving the quality of life of GSD-I patients, and playing a vital role in funding research that not
                                                                                                                                                                          by The Andrushko’s
                                                                                                                                                                                                    In July of this year we held the “6th Annual   We could not have asked for a better day - the sun was shining, it
                                                                                                                                                                                                    BBQ Fundraiser for GSD Research” in            was hot outside, and there was so much to do! From great food &
 Dr. Dwight Koeberl.                                     fasting at 5 months of age. In summary, long-term        original virus (the chicken beta-actin promoter                                   honor of our little boy Kasen. This day        music, to bouncers & ponies - everyone was entertained - over 200
 Division of Medical                                     efficacy of AAV vector-mediated gene therapy was          and CMV enhancer). More importantly, we have                                      went way beyond our expectations! We           of you!
 Genetics/Department                                     demonstrated by the correction of hypoglycemia           shown that this new virus can still effectively                                   were able to raise over $18,400 - the          A special thank you to the Ultimate Canadian Motorsports
 of Pediatrics, Duke                                     and lactic acidemia during prolonged fasting in          correct the GSD-Ia defects in the mouse model.                                    most to date totalling over $70,000            Association who have joined out fight for a cure! In honor of Kasen
 University Medical                                      canine GSD Ia.                                           Indeed when we compared the efficacy of liver                                      raised over the past six years at our          Andrushko, the organization has shown their support and we have
 Center, Durham, NC                                                                                               G6Pase delivery of our new virus, to the previous
                                                                                                                                                                                                    event. The families and friends of all         raised over $1000 and counting by them allowing us to raffle off
 In response to an                                                              Janice Chou, Ph.D.                version of the virus, we showed that the new virus     Kasen Andrushko
                                                                                                                  not only directs more efficient expression but also                               the GSD families involved in Manitoba           items at their motorsports events. Please check out the website at
 invitation to provide                                                          National Institute of Health
 information regarding our research funded by The                               We have identified the gene        more persistent G6Pase expression in the liver than    are one of a kind. We truly appreciate the kindness you have all for more events to come! Thank you Jake & Lisa Funk!
 Children’s Fund for Glycogen Storage Disease,                                  for the G6Pase enzyme             the previous virus.                                    shown us for our fight to finding a cure for these precious kids.
 I wish to provide the update regarding our gene                                and generated a mouse             Our work has established two key milestones in GSD-
 therapy research. The canine GSD Ia model has                                  model of GSD-Ia to increase       Ia research. Firstly, unlike the old virus, the new
 features similar to human GSD Ia that are highly
 relevant to the development of a new therapy,
                                                                                our understanding of the
                                                         pathogenesis of this disorder and to develop
                                                                                                                  virus expresses G6Pase activity in the liver of GSD-
                                                                                                                  Ia mice as effectively as the natural G6Pase gene
                                                                                                                                                                         Shooting for a Cure                                                 A Taste of Delray!
 including profound hypoglycemia upon fasting            new therapies. Using the mouse model, we have            in wild-type mice at age 6 months. Secondly, these     by Stacey Becker                                                    by Jennifer Mills
 and other biochemical parameters, glucose-6-            developed a promising gene therapy that uses a           levels of expression are therapeutic, with the new                                                                         On December 4, 2008, the parents of Zoey
 phosphatase deficiency in the liver and kidney           modified adeno-associated viruses (AAV) vector            virus-treated GSD-Ia mice no longer suffering from     My fiancé, Jonathan Wanicur and I joined an organization             Robinson, (GSD type 1a) hosted “A Taste
 accompanied by glycogen accumulation in those           where G6Pase is controlled by the commonly used          fasting hypoglycemia. Indeed, the blood glucose        called Zogsports in New York City, which runs adult sports          of Delray” in Delray Beach, Florida. It
 tissues, and reduced survival. Treatment with           regulator from chicken-actin promoter and a              levels in the treated GSD-Ia mice were unchanged
                                                                                                                                                                         leagues in every sport imaginable. Zogsports, whose motto           was a cocktail reception featuring amazing
 an adeno-associated virus type 8 pseudotyped            powerful CMV enhancer to deliver the G6Pase gene         after 6 hours of fasting, indistinguishable from
                                                                                                                                                                         is “Play for Your Cause,” has each team choose its favorite         culinary delights from some of the area’s
 vector (rAAV8) has prolonged the survival of all        into a GSD-Ia mouse. We have shown that using            that in the wild-type mice.
 three GSD Ia dogs to >12 months, in contrast to         this modified vector, we can deliver a good copy of       What remains to be determined is if the changes        charity. At the end of each season, the winning team’s charity      best restaurants. There was a silent auction,
 the abbreviated survival of untreated GSD Ia dogs.      the G6Pase gene into the liver of a GSD-Ia mouse,        in our new virus will also reduce the incidence of     receives a check. The latest and greatest league to come out        a Wheel of Hope, where you got to spin and
 Vector-treated GSD Ia dogs thrived, no longer           where it expresses the G6Pase protein and corrects       gene therapy-induced hepatocellular carcinoma          this past winter: adult, co-ed floor hockey, and we decided to       win some great prizes, and a live painting
 requiring carbohydrate supplementation following        the GSD-Ia defects. This suggests that gene              or not, and how long the introduced gene will          give it a try.                                                      that was raffled off at the end of the night.                  Zoey Robinson
 the newborn period and were fed only three times        therapy could be a way to treat the disease in GSD-      continue to function efficiently. These issues must     We named our team “Two for Slashing.” Jonathan Wanicur              Thank you to Delux Nightclub for doing an
 daily, every 8 to 10 hours, while untreated GSD Ia      Ia patients, but more research needs to be done          be further investigated before AAV-mediated gene       and Dion DeVries were co-captains of our team, and chose            amazing job sponsoring the event! Sandy and
 dogs previously required very frequent feedings         before we can try this approach with patients.           therapy in human GSD-Ia could be contemplated.         The Children’s Fund for GSD Research to be its charity, after I     Lisa Hodes were there to help as well as some other fabulous volunteers.
 of a high carbohydrate diet. Two of three GSD Ia        Prior to any experimentation with human gene             We are now examining the long-term consequences
                                                                                                                                                                         informed the team about my nephew, Jonah Feldman, and all           It turned out to be a great success! They raised $6,650 for The Children’s
 dogs had normal glucose levels following 6 hours        therapy, we have to improve the safety of the            of our new virus-mediated gene therapy using the
 of fasting at 14 and 16 months of age, surpassing       virus used in gene therapy and make sure the             GSD-Ia mice. The goal of our study is to generate
                                                                                                                                                                         the wonderful work being done by the foundation.                    Fund for Glycogen Storage Disease. They look forward to next year’s event
 the degree and duration of efficacy previously           G6Pase gene is expressed correctly. Funded by            an AAV virus that possesses the best safety            Thank you to our fellow members of “Two for Slashing”: Dion         and raising even more money to find a cure for GSD!
 reported following gene therapy in GSD Ia dogs.         The Children’s Fund for GSD Research, we have            profile as a candidate for future clinical gene         DeVries, John Saroff, Michael Palan, Piper Loving, Sean
                                                                                                                                                                         Stevens, Alex Gagnon, Alex Pugatch, Zach Abramowitz,
 These latter two GSD Ia dogs, Risk and Lollipop,
 continue to thrive with normal care, completely
                                                         generated a safer, more effective version of the
                                                         AAV virus. In most gene therapy studies, to get
                                                                                                                  therapy trials for human GSD-Ia patients.
                                                                                                                                                                         Alana Benjamin, Paul Benjamin, Geetha GoPalan, Laura
                                                                                                                                                                                                                                             EMS Bike Ride for GSD
                                                         the best level of expression of the gene being used,
                                                                                                                                                                                                                                             The Belot Family
 asymptomatic at >two years of age. Lactic                                                                                        Howard H. Sussman, M.D.                Guy, Simon Chan and Chris Larkin for choosing The Children’s
 acidosis reflects poor metabolic control and a risk      researchers use very powerful gene promoters and                         Stamford University                    Fund and spreading awareness of GSD on behalf of Jonah
 for renal complications in GSD Ia, and therefore        enhancers, like the chicken beta-actin promoter                          After 10-20 years about 50%                                                                                Brent Belot, father of Jessie who has GSD 1a
                                                                                                                                                                         Feldman, and all children and their families everywhere
 the correction of lactic acidosis is an important       and CMV viral enhancer used in our study. However,                       of GSD1a patients develop              affected by GSD. Let’s get ready for next season!                   works for the Winnipeg Fire and Paramedic
 endpoint for therapy in GSD Ia. Plasma lactate          despite the fact that the AAV-based vectors, like the                    hepatic adenomas from which                                                                                Service. He helped to organize this years
 during fasting was reduced for vector-treated           one we use, have the best safety profiles among the                       many succumb to hemorrhages                                                                                ‘Emergency Medical Services Bike Ride.’ The Jessie Belot and supporting
 GSD Ia dogs at one month of age, equivalent to          viruses widely used in gene therapy, a recent study      because the hepatic adenomas affect the clotting
 the level observed for carrier dogs. Plasma lactate     showed that neonatal mice treated with an AAV                                                                                                                                       event raised $1,340.00 for The Children’s friends from the Winnipeg Fire
 levels have normalized at 14 and 16 months of           vector expressing a human gene directed by the           The basis for the development of the hepatic                                                                               Fund. During the Bike Ride, Northpride and Paramedic Service
 age for Risk and Lollipop. We interpret this data       regulator, chicken -actin promoter/CMV enhancer,         adenomas is not known. The objectives of the                                                                               Sportswear raised $70.00
 as evidence for stable transgene expression             have an increased incidence of hepatocellular            present investigation are to identify genes that                                                                           from the sale of t-shirts
 and decreasing glucose requirements with age.           carcinoma. To address the possibility that the           are expressed in the liver of patients with GSD1a                                                                          and shorts. We all had a
 Furthermore, recent experiments with an rAAV9           non-human gene controls are part of the cause of         which will provide us with molecular markers for
 vector revealed correction of hypoglycemia. The         hepatocellular carcinoma, we identified the native                                                                                                                                   great time and very much
                                                                                                                  understanding the basis for the risk of developing
 first GSD Ia dog treated with this rAAV9 vector          human G6Pase gene regulator and enhancer and             hepatic adenomas and for use in identifying the                                                                            enjoyed the day!
 had normal blood glucose throughout >6 hours of         used them to replace the non-human parts of the          transformation to the adenoma state. The goal is

 2                                                                                                                                                                                                                                                                         7
The Race to Cure GSD                                      Intense Training’s 5k Challenge for a Cure by Lisa Hodes                                                           Sc i e n t i f i c U pda t e. . .
                                                              Northwest Broward Roadrunners Club who          organize an event or participate individually     to identify a genetically recognizable subset of    to determine pathways that may be involved                             Dr. Thomas Conlon , University of
                                                              helped to make sure this inaugural event        in your area, please contact Lisa Hodes at        patients who develop hepatic adenomas. The          in this transition. We will be conducting                              Florida
                                                              was a success. With your support, we raised or 954-263-1631. A               hope is that in this patient subset it will be      micro-array tests on microRNAs from the                                Glycogen storage disease type Ia is
                                                              about $6,000 for GSD Research. A special        pair of sneakers and some motivation is all       possible to predict the transition to hepatic       same specimens that were used for the                                  caused by a deficiency in glucose-6-
                                                              shout out to Brian and Desiree Makovsky of      you need to help find a cure for our children.     adenoma.                                            genomic RNA analyses, as described above.                              phosphatase. GSDIa patients are unable
                                                                                                                                                                Our studies have been directed to this goal         MicroRNAs are a species of RNA that regulate                           to maintain glucose homeostasis and
                                                              Intense Training for being our title sponsor    Run on…
                                                                                                                                                                to identify molecular markers that may              gene expression.                                     suffer from severe low blood sugar and an enlarged liver.
                                                              and for their unending and ever-present                                                           be indicative of the transition to hepatic          Also to be noted is that we have increased           The canine model of GSDIa is naturally occurring, and
Jena Piekarski, Denise Harrison, Lisa Hodes, Kim Harris       support. We truly appreciate all you do for                                                       adenoma. Experiments using micro-array              the number of GSD1a liver specimens for              recapitulates almost all aspects of the human form of
Our first ever Race to Cure GSD took                           our family and all others who are affected by                                                     methods that identify approximately 44,000          analysis, as well as obtaining an increase           disease. We investigated the potential of recombinant
place on June 14th at Quiet Waters Park in                    Glycogen Storage Disease.                                                                         genes that are either up-or down regulated          in non GSD1a liver specimens. The addition           adeno-associated virus vector-based therapy to treat the
                                                              Team Intensity’s next race to support GSD                                                         in GSD1a in association with transition to          of more specimens increases the statistical          canine model of GSDIa. After delivery of a therapeutic
Deerfield Beach, FL. About 200 people from                                                                                                                                                                                                                                gene therapy vector to a one-day-old GSDIa dog named
                                                              Awareness will be the Walt Disney World Half-                                                     hepatic adenoma have been completed. These          value of the data.
the ages of 7 to 79 came out to participate                                                                                                                     data are being analyzed at the present time                                                              “Dulce”, improvement was noted as early as 2 weeks
in the 5K run/walk in support of Glycogen                     Marathon (13.1 miles) on January 9, 2010 in
                                                                                                                                                                                                                                                                         post-treatment. Correction was transient, however, and
Storage Disease. Medals and trophies                          Orlando, FL. Please support our efforts by                                                                        Dr David Weinstein,                 2006 – 2007 using grant funding provided             the treated dog could no longer sustain normal blood
were awarded to all of our top finishers, but                  visiting, clicking on Team                                                                        University of Florida and           by the Children’s Fund for GSD Research. In          glucose levels after one hour of fasting by two months
we congratulate everyone who completed                        Intensity and making a donation. We will        Team NBPS                                                         Dr. Priya S. Kishnani, Duke         a pilot study of 12 children, the new product        after treatment. The same animal was then dosed again,
the 3.1 mile course. Thanks to all of our                     not stop running until we find a cure!                                                                             University Medical Center           was found to last several hours longer than the      but delivered directly into the liver’s circulation. Two
                                                              If you would like to join Team Intensity and                                                                                                          conventional therapy, and it had an improved         months after the second dose of gene therapy, both
sponsors, in-kind donors, friends and the
                                                                                                                                                                                 Although cornstarch therapy        safety profile since the rate of glucose fall was     blood glucose and lactate levels were normal at 4 hrs
                                                                                                                                                                                 has dramatically improved          much slower even after hypoglycemia develops.        after food was withdrawn. With more prolonged fasting,
Awesome GSD Fundraising Events in Honor of Rylee Graham!!                                                                                                                        the quality of life for patients   The results from this study were published in        the dog still maintained normal glucose concentrations,
                                                                                                                                                                                 with glycogen storage disease      the American Journal of Clinical Nutrition.          but lactate levels were elevated by 9 hours, indicating
                                              This past summer, a golf                Rylee’s brother Reese had a birthday and asked for donations to be                                                            While the new product offers hope that people        that partial correction was achieved. All dietary glucose
                                                                                                                                                                                 type I, it is far from an ideal
                                              tournament was held in                  sent to our foundation. He raised $110 in March 2009. Thank you                            treatment. Even the highest        with GSD will be able to sleep through the night     supplementation was discontinued starting at one month
                                              Martinsville, Virginia in               and Happy Birthday!                                                       quality cornstarch only prevents hypoglycemia       in the future, a second study was needed to          after the second dose delivery, and the dog continues
                                              honor of Rylee, GSD 1a.                                                                                           for a median time of 4.25 hours in children,        learn how the new product should be optimally        to thrive with minimal lab abnormalities at 25 months
                                              We would like to take this                                       In January 2009, the children of Ilene and       and all children (or the parents) must awaken       dosed. The 2 major GSD institutions in the           of age. These results demonstrate that delivery of our
                                              opportunity     to commend                                       Donald Newman of Spencer, VA – Kathy             in the middle of the night to administer the        United States are collaborating on this work, and    gene therapy vectors results in significant correction of
Rylee with her Great Aunt Lisa, bother Reese
                                             the Lynwood Ladies Golf                                           Graham, Debbie Simmons and Eddie                 life sustaining therapy. Even in adulthood,         investigations began in 2008 on 60 children and      the GSDIa symptoms, and that this strategy may be a
and cousins, Lyndsey and Mason
                                             Association at Lynwood                                            Newman threw their parents a surprise 50th       few people can make it through the night, and       adults with type Ia GSD. To date, approximately      promising alternative therapy for this disease or other
                                                                                                                                                                most people with glycogen storage disease           35 subjects have been studied as part of the         forms of glycogen storage disease.
 Country Club as well as all of the fine women who participated in                                              anniversary party. They requested that gifts
                                                                                                                                                                never obtain a full night of sleep. Yet, people     second dosing study, and investigations should       Additionally, we have recently shown that our gene
 the golf tournament. The tournament raised $1500 for The Children’s                                           be made to The Children’s Fund in their honor,                                                                                                            therapy provided enough correction of the disorder in
                                                                                                                                                                with GSD are dependent upon receiving the           be completed by 2010. Because the study is
 Fund for Glycogen Storage Disease Research. Rylee’s great-aunt,                                               to help one of their grandchildren, Rylee                                                                                                                 “Dulce” to allow her reproductive organ systems to fully
                                                                                                                                                                cornstarch on time. Delayed administration of       blinded (to both the investigator and subject),
 Lisa West, is a member of the Lynwood Ladies Golf Association                                                 Graham who has GSD1a. Their celebration                                                                                                                   develop to the extent that she could have puppies. This
                                                                                                                                                                the cornstarch is associated with development       it is not known at this point how the 2 starches
 and brought The Children’s Fund to the association’s attention.                                               raised $1,600.                                                                                       compare or which dose will be best. We remain        finding is extremely exciting to us as it has not been
                                                                                                                                                                of hypoglycemia which can result in seizures,
 This was our first experience with this type of fundraiser. We                                                                                                  brain injury, and even death. The constant          hopeful that the new starch will allow patients      shown that animals with this disease are able to carry
 cannot tell you how incredibly grateful we are to everyone                           Thank you to the staff at Huntsville Elementary School in Madison,                                                            to maintain normal blood glucose levels for          a litter. Finally, we have developed a new method of
                                                                                                                                                                anxiety associated with living by the clock
 who helped and for all of the gracious donations. We are                             NC. They participate in ‘Huntsville Gives Back’ each month when                                                               longer periods of time.                              watching the progression of the disease, results of gene
                                                                                                                                                                and the need to interrupt sleep 1-3 times per
 confident that GSD can be cured in the near future .... We’re                         they donate money to wear jeans the first Friday of each month and                                                             The Children’s Fund for GSD Research continues       therapy and possible formation of Adenomas through
                                                                                                                                                                night is deeply detrimental for the children and
                                                                                                                                                                                                                                                                         Magnetic Resonance Imaging and Spectroscopy (MRI/
 grateful to the Lynwood Ladies for helping us in our endeavors.                      the money is sent to a deserving charity. The Children’s Fund was         their families. The effort is exhausting, and       to be the only source of funding for this work. As
                                                                                                                                                                                                                                                                         MRS) in a live dog or patient without having to take a
                                                                                      chosen for the month of April and they raised $265.00!! This is the       eventually delayed administration of cornstarch     a result of the investigations completed to date,
                                                                                                                                                                                                                                                                         liver sample.
Thank you to the Western Rockingham Junior Service League,                            most money they have raised in a single month – It was in honor of        occurs due to the unavoidable fatigue.              approval for the use of the modified cornstarch
                                                                                                                                                                                                                                                                         Since, we have increased the size of our group of GSD1a
a community service organization which helps to raise money for                       Rylee Graham who attends the school and her mom, Kathy who works          In 2003, the need for improved therapies was        has been obtained in England, Australia, and
                                                                                                                                                                                                                                                                         dogs to include additional GSD diseased animals and a
                                                                                      there!                                                                    emphasized by the parent representatives            throughout most of Europe. Approval in the
charitable organizations. This year they chose to give our foundation                                                                                                                                                                                                    group of animals that are carriers for the disease as well
                                                                                                                                                                at the first Children’s Fund for GSD Research        United States and Canada is dependent upon
$250 in honor of Rylee Graham.                                                                                                                                                                                                                                           as normal dogs. Data is being collected in upwards of 20
                                                                                                                                                                symposium. A task force was created, and            improved dosing information.
                                                                                      Thank you to Racing 2 Save Lives – The Children’s Fund was awarded                                                                                                                 animals in order to track the progression of the disorder
                                                                                                                                                                multiple products were tested over the past         For more information about the study, please
                                    Zeb Williams and Lexi Blackard of North           $4,000 as a result of the 2008 fundraising efforts in honor of Rylee.                                                                                                              as a model of the human form in comparison to people
                                                                                                                                                                5 years. Through the help of Dr. Philip Lee         contact Anne Boney at Duke University (919-
                                    Carolina had a combined 12th birthday                                                                                                                                                                                                without the GSD. The significant amount of data will
                                                                                                                                                                in England, an outstanding new sustained            681-1932) or Laurie Fiske at the University of
                                    party to raise money for The Children’s                                                                                                                                                                                              help support an Investigational New Drug submission
                                                                                                                                                                release cornstarch preparation was identified,       Florida (352-273-6655).
                                                                                                                                                                                                                                                                         for the FDA to review in an effort towards moving this
                                    Fund in honor of their friend, Rylee. The                                                                                   and the product underwent initial testing in                                                             therapy to the clinic.
                                    party raised $1,100. WOW!!

6                                                                                                                                                                                                                                                                                   3
 Fall 2009                                        GSD In The News
                                This past holiday season, Ally                                            This past year, 3 friends did 3 very
                                                                                                                                                                                                    Volume 7
                                                                                                                                                                         In April the Student Council at North
                                                                                                                                                                                                                                     GSD raffle for Christmas Cash!!
                                                                                                                                                                                                                                     by Donna McNeil
                                                                                                                                                                                                                                     Thank you so much to Donna
                                                                                                                                                                                                                                     McNeil for organizing a
                                Feldman of Cheshire, CT – asked that                                      special things. Danny Rosen of                                 Broward Preparatory School in Coral                         wonderful raffle held on
                                her Secret Santa make a donation to                                       Katonah, NY celebrated his Bar                                 Springs, FL elected The Children’s Fund,                    Dec. 6, 2008 in honor of her
                                GSD instead of receiving a gift. She                                      Mitzvah and asked his guests to                                along with 2 other charities, to be a                       granddaughter,        Kamryn
                                wants to make the world a better                                          contribute to The Children’s Fund for                          beneficiary of a dress down day. Students                    Jackson. The raffle was for
                                place by helping to find a cure for her                                    GSD Research instead of receiving        Mrs. Malanowski and   who brought a minimum donation of $2                        $1,000 cash and it raised
                                cousin, Jonah. She also gave up her                                       his own gifts. He was able to raise      Samantha Hodes        were given permission to wear attire other                  $18,450 for GSD research!
                                                                                                                                                                                                                                                                                Kamryn and family with winner Jack Farrell
                                last night of Hanukah presents and       Chase Madorsky, Dani Josephson almost $6,000 for the foundation.          than their usual school uniform. A total of $300 was raised for                   A special thanks goes to
                                made a donation to The Children’s        and Danny Rosen                  Chase Madorsky of Livingston, NJ         GSD Research. Thanks to Kathleen Malanowski, Licia Campbell,                      Kamryn, Rylee and Taner,
                                Fund. She truly knows the meaning                                         chose GSD as his Mitzvah project for     the Student Council officers, and all who participated for their                   who went around the neighborhood selling tickets, raising $210.
Ally and Jonah Feldman                                                   his Bar Mitzvah and had a garage sale in the pouring rain that raised     support.                                                                          The way it worked: It was a very easy way to raise money with very little
                                of the holiday season! Thank you
                                                                         $800. And, most recently, Dani Josephson of Jericho, NY generously                                                                                          work. I simply wrote a letter to all the friends and family who have helped
                                                                         and unexpectedly contributed to our charity on her Bat Mitzvah day.       ‘Shopping for the Holidays’ in Florida                                            us in the past, with Kamryn’s picture and an update on her condition
In March 2009, Letty King of Tucson,                                     All of these kids did this because they want to make a difference for     raised almost $800 for GSD Research                                               and told them about the raffle. The response was great!
AZ decided to do some spring cleaning                                    Jonah Feldman and kids like him. Danny, Chase and Dani have grown         with a holiday boutique – Each vendor                                             The raffle was held just before Christmas – who couldn’t use $1,000
and had a huge garage sale. She                                          up together, and now go to the same summer camp where their fathers       donated a portion of their proceeds to GSD                                        for their holiday shopping? Tickets were sold for $10 each. We
donated all of the proceeds to The                                       met Jonah’s dad when they were kids at Camp Scatico in Elizaville, NY.    and we all walked away with new jewels,                                           were going to take the $1,000 out of the proceeds, but God Bless
Children’s Fund in honor of her                                          The values that each of these families has instilled in their children    purses and gifts for the holidays. Thanks                                         Jack Farrell, the man who won. He bought $1,000 worth of tickets
grandson, Max Blechman! Thank you                                        are truly amazing. The Feldmans are so touched by their generosity        to Grace Surdis for organizing the event                                          then gave his winnings back to us. When we took the picture for the
so much.                                                                 and for their willingness to spread the word about our cause to their     and hosting at her house and to all the             The Hodes Family              newspaper and presented him with his money, Jack turned to Kamryn
                                                                         friends and families. We love you guys!!                                  vendors who so generously donated to our                                          and said “Now that that is done, you take this and use it for finding
Thank you to everyone who participated Max Blechman & Letty King                                                                                   cause. We look forward to another girls night in a few months!!                   a cure for you and all those other precious kids.” So everything we
                                                                                                         In February of 2009, Elizabeth Perez                                                                                        made was profit for the raffle. I am sure not everyone would have
in the 2009 Super Bowl Pool for GSD                                                                      organized a fundraiser in Texas for her                                                                                     done this. I always feel God has a hand in matters such as this.
run by the Casarella family of Southington, CT once again!                                               son, Ryan Salinas. The party went well    Thank you to all who participated in Spa Day for GSD at Simply Me                 I am so glad our family can be a part of finding a cure for GSD.
Congratulations to winners: Marc Jacobson, Holly Blanchette,                                             – the clown and moonwalk were lots        Day Spa in Coral Springs, FL on behalf of Sam and Katie Hodes.                    We are passionate about raising money to help with this cause.
Dennis Rodin, Ken Casarella, Larry Fiedler, Bill Gould, Charlie                                          of fun! Over $400 was raised for GSD      $200 was raised!                                                                  Thank you for everything you do to make this possible.
Hague and Natale DiNatale. This year’s pool raised $3,500!!                                              Research. Thank you so much to everyone
                                                                                                         who was there!                            2nd Annual Band for the Cure
                                         Great job Jack and Anna
                                         Reed and their cousins
                                                                         Ryan Salinas and his parents.                                             by Jeanne Muchnick                                                                Kure for Kamryn Bike-a-thon!!
                                         Timothy, Zachary and Chris                           In 2009 we lost someone very near and dear to one    The music was jamming, the
                                                                                                                                                                                                                                                               Kamryn Jackson’s preschool, The Christian
                                         Ploch of New Canaan, CT!                             of our GSD families, Mr. Donald Bagin, grandfather   conversation enjoyable and the
                                                                                                                                                                                                                                                               Learning Center at the First Baptist Church in
                                         Their lemonade stand raised                          of Justin Bagin. Our foundation received so many     silent auction quite busy at the
                                                                                                                                                                                                                                                               Canyon, TX had a Bike-a-thon to help raise money
                                                                                              contributions made in his memory. Mr. Bagin          ‘Second Annual Band for the
                                         $55.35 for GSD Research!                                                                                                                                                                                              for “Kure for Kamryn” and The Children’s Fund for
                                                                                              even requested that The Children’s Fund be gifted    Cure’, organized by GSD mom                     Singers Elza, Michael Nappi and                             GSD Research. Mrs. Susan Young is the school
Thank you to Girl Scout Troup #64088 of Chase Collegiate                                      $15,000 from his estate. In total, almost $28,000    Jeanne Muchnick (daughter is                                   Jeanne Muchnick                              director, and she is very interested in helping us
School. They donated $150 to our foundation from money raised                                 was received to help his grandson and others         Corey Rosen). The event was held
                                                                                              born with GSD1a. We send our condolences to the                                                                                                                  raise money and awareness for GSD.
by selling Girl Scout Cookies. The members of the troop are all                                                                                    at The Globe Restaurant in Larchmont which generously donated their
                                                                         Justin Bagin         Bagin family and our thanks for all of the support                                                                                                               The children brought their bikes and helmets to
friends with Ally Feldman and they know how committed she is to                                                                                    space. Singers Elza ( and Michael Nappi (www.
                                                                                              during this difficult time.                                                                                                                                       school. Mrs. Young and the staff blocked off the
a cure that would help Jonah and others with this disease.                                                                               , musicians with cd’s and a long list of accolades,
                                                                                                                                                                                                                                                               parking lot, placed cones to form a race track, and
                                                                         Happy 2nd Birthday to Henry Solomon of Atlanta, GA – his friends          also donated their time and energy to create a coffee shop atmosphere,
A special thank you goes to Mr. Richard Iagrosse of Northford, CT                                                                                                                                                                                              the children rode their bikes for about an hour.
                                                                         and family donated to our foundation at his party in honor of Jonah       complete with wine, food and amazing silent auction items donated
                                                                                                                                                                                                                                                               Everyone had a great time, especially Kamryn.
whose generosity is truly appreciated!                                   Feldman! How sweet!!                                                      mostly by Westchester-area stores and businesses. The event raised
                         Thank you to Tom O’Day, friend of the Reed                                                                                $5,885.00, all in an effort to “band together” and find a cure for GSD.
                                                                         A special thank you to Kathy Turi, mom of Julianna, who has been          “I’m constantly amazed by the warmth and support of the community,”
                         family for making a splash at the New
                         Canaan Sidewalk Sale. He sat in a dunking
                                                                         working hard to spread the word about, a website that
                                                                         donates $ back to our cause when you make purchases!
                                                                                                                                                   says Jeanne, “Especially when you open up and tell them about what
                                                                                                                                                   life is really like with a GSD kid. To be honest, I was afraid to do this
                                                                                                                                                                                                                                       The 5th Annual GSD Golf Classic
                         booth for an hour and raised $278 for us!                                                                                 event in this economy…I didn’t think I’d be able to raise much money...              will be held in Florida on May 7th 2010.
                                                                         Thank you to Sue Wolff who continues to speak on our behalf to The
                                                                                                                                                   but I exceeded my goal. To me, every little bit counts and I’m happy for              Please visit for details!
                                                                         Optimist Club of Winston-Salem about our cause. We appreciate
                         Tom O’Day and Family                                                                                                      whatever we raise.”
                                                                         your efforts!

4                                                                                                                                                                                                                                                                          5

To top