Avery’s Hope

Avery’s Hope Fund was established to raise public awareness about Glycogen Storage Disease (GSD) and to support fundraising efforts to find a cure for this disease.

Glycogen Storage Disease Research Fund

Specifically, Avery’s Hope supports the research efforts of Dr. David Weinstein’s Glycogen Storage Disease Program at Connecticut Children’s Medical Center.

100% of all monies raised by Avery’s Hope will be used to support research projects related to the treatment and cure of Glycogen Storage Disease.

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Pompe Gene Therapy Found Safe During Human Trial

From UF Health A gene therapy that treats respiratory problems in early-onset Pompe disease was shown to be safe during its first human trial, University of Florida Health researchers have found. The inherited disease causes a complex sugar to accumulate in cells,...

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Glycogen Storage Disease

Glycogen Storage Disease

Glycogen Storage Diseases (GSDs) are a group of rare inherited genetic disorders caused by an inborn error of the metabolism. Those affected with GSD have a defective gene that results in an enzyme deficiency that alters the ability of the body to make energy during periods of fasting.

Metabolism is the combination of all the chemical reactions taking place in the body to convert or use energy. It is the process by which our body breaks down the food that we eat and converts it to the energy that is used by the body. When just one enzyme that make these chemical reactions possible is missing or damaged, the entire metabolism process is disrupted.

Normally, when a person eats, excess sugars that are not immediately used by the body, are stored in the liver and muscles as glycogen. During periods of fasting, the liver releases the stored sugar to provide energy for the body. In the case of GSD, because of the missing enzymes, the body cannot make enough glucose or is not be able to use glucose as a form of energy, the stored glycogen begins to build up in the organs, and the body cannot maintain normal blood sugar levels without constant feedings.

 

At this time there is no cure for GSD.

Dangerous by-products such as lactate, uric acids,and triglycerides in combination with low blood sugars lead to numerous complications including impaired breathing, seizures, coma and death if a very strict dietary regimen is not followed. The management of GSD is lifelong and not only affect the child, but also affects the parents and family of the child born with this disease. These children must be fed every one to four hours to maintain appropriate blood glucose levels. Because this regimen of constantly feeding day and night and because missing a meal or feeding can have catastrophic effects, some children have gastric tubes placed and some of these have issues ingesting by mouth.

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Director, Glycogen Storage Disease Program

 

DR. WEINSTEIN

At present, all of Dr. Weinstein’s research expenses are being funded by philanthropy from individuals and groups such as Avery’s Hope Fund.

Weinstein’s GSD Program,  is the largest clinical and research program of its kind in the world. Pediatric and adult patients living with the rare, genetic liver disease travel from across the globe for his team’s expert care.

Weinstein graduated from Connecticut’s Trinity College and earned his medical degree at Harvard Medical School. At Boston Children’s Hospital he completed his residency, chief residency, and fellowship in pediatric endocrinology and completed his masters in clinical investigation at Harvard and MIT. He became the director of the GSD program at Boston Children’s before moving to Florida in 2005 to pursue gene therapy on dogs with naturally occurring GSD.

Prior to joining UConn Health, Weinstein served as professor of the Division of Pediatric Endocrinology at the University of Florida and director of its GSD Program. As a physician-scientist he has authored over 80 articles and 26 textbook chapters on GSD. He is the recipient of the prestigious international humanitarian award, the Order of the Smile, for helping children around the world with GSD, a recognition shared with Pope John Paul II, Mother Teresa, Nelson Mandela, and Pope Francis. Weinstein was named one of the inaugural Goldwater Scholars in 1989. He is a former Jan Albrecht Award winner from the American Association for the Study of Liver Diseases, and he received the George Sacher Award from the Gerontological Society of America. Weinstein was inducted into the Rare Disease Research Hall of Fame in 2013. In addition to local GSD community outreach, Weinstein’s team partners internationally with institutions and scientists in Argentina, Brazil, Canada, China, Faroe Islands, Israel, Mexico, and the Netherlands.

After nearly two decades of dedicated research, Dr. Weinstein will begin human safety trials of his gene therapy. Expected to start in early 2018, UConn will coordinate the trials with collaborating centers all over the world

Director of Glycogen Storage Disease Program

DR. WEINSTEIN

Weinstein’s GSD Program,  is the largest clinical and research program of its kind in the world. Pediatric and adult patients living with the rare, genetic liver disease travel from across the globe for his team’s expert care.

Weinstein graduated from Connecticut’s Trinity College and earned his medical degree at Harvard Medical School. At Boston Children’s Hospital he completed his residency, chief residency, and fellowship in pediatric endocrinology and completed his masters in clinical investigation at Harvard and MIT. He became the director of the GSD program at Boston Children’s before moving to Florida in 2005 to pursue gene therapy on dogs with naturally occurring GSD.

Prior to joining UConn Health, Weinstein served as professor of the Division of Pediatric Endocrinology at the University of Florida and director of its GSD Program. As a physician-scientist he has authored over 80 articles and 26 textbook chapters on GSD. He is the recipient of the prestigious international humanitarian award, the Order of the Smile, for helping children around the world with GSD, a recognition shared with Pope John Paul II, Mother Teresa, Nelson Mandela, and Pope Francis. Weinstein was named one of the inaugural Goldwater Scholars in 1989. He is a former Jan Albrecht Award winner from the American Association for the Study of Liver Diseases, and he received the George Sacher Award from the Gerontological Society of America. Weinstein was inducted into the Rare Disease Research Hall of Fame in 2013. In addition to local GSD community outreach, Weinstein’s team partners internationally with institutions and scientists in Argentina, Brazil, Canada, China, Faroe Islands, Israel, Mexico, and the Netherlands.

After nearly two decades of dedicated research, Dr. Weinstein will begin human safety trials of his gene therapy. Expected to start in early 2018, UConn will coordinate the trials with collaborating centers all over the world

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