GSD III Study

The Johnny Damon Foundation proudly supports GSD Research and David Weinstein, M.D., M.M.Sc. who is the Director and Professor of Pediatric Endocrinology at Shands Hospital at the University of Florida.

Glycogen Storage Disease (GSD) was almost universally fatal until 1971 when the first effective therapy was discovered, cornstarch. While this may not sound sophisticated, it turned this from a fatal disease to one where people are thriving. When treated properly, patients are healthy; when suboptimal treatment occurs, however, patients may develop permanent neurologic damage, seizures, or even die. Even with modern therapy, complications remain common including hepatic adenomas, liver cancer, renal failure, osteoporosis, anemia, and inflammatory blowel disease.

In 2005, Dr. Weinstein moved from Harvard Medical School and Children's Hospital Boston to the University of Florida in order to pursue a dream of performing gene therapy in glycogen storage disease. The lack of a vet school in Boston made it impossible to perform the studies, and the University of Florida offered outstanding research infrastructure. Since arriving 5 years ago, core teams have been created in 10 areas.

  1. Gene Therapy Team
  2. Adult Stem Cell Team
  3. Nerologic Disorders and Attention Deficit Hyperactivity Team
  4. Treatment Trial Team
  5. Cardiovascular Team
  6. Exercise Team
  7. Anemia Team
  8. Inflammatory Bowel Disease Team
  9. Liver Team
  10. Dental Research Team

 

We are very excited to be involved with DR. Weinstein's Faroe Islands Research Project.

The research helped stimulate interest in the Faroe Islands (where 1 in 800 people have the disease), and helped create the foundation for the new type III research institute.

  The new research institute has several goals:

           To study the interventions made in this population which may provide clues regarding ways to better treat theGSD III population

           To provide a local facility to facilitate GSD III research

           To investigate the possible link between carrier status and muscle disease

 

Here are some images of the from Dr. Weinstein's latest visit to the Island.