A Danon Disease Study Wins Top Research Award
The Top 10 Clinical Research Achievement Awards recognize important breakthroughs in clinical research from across the United States. The awards highlight major advances in health and medicine made possible by the nation's investment in research. Winners were recently chosen by a panel from the Clinical Research Forum based on studies published in peer-reviewed journals in 2025. The panel looked for innovative research that improves understanding of human health and disease, and that has the potential to improve how diseases are diagnosed, prevented, and treated.
We are happy to share some good news for patients and families living with Danon Disease: one of the award-winning studies was in this rare disease! The study, led by researcher Dr. Barry Greenberg and published in the New England Journal of Medicine, focuses on gene therapy as a potential treatment option for Danon disease. The study showed early signs that gene therapy may help the hearts of people with Danon disease.
“This study shows that it may be possible to deliver healthy copies of a faulty gene to the hearts of patients with cardiomyopathy, a disease that weakens the heart muscle,” said Greenberg. “The treatment was linked to improvements in the structure of the heart. Patients who received the therapy also showed stable or improved health. Based on these results, a larger clinical trial is now underway.”
If you or your loved one is living with Danon disease and would like to learn more about the latest research opportunities in your area, please fill out this quick form:
