- recruiting
Danon Disease Natural History Study
This study aims to understand Danon disease better by looking at health information from people living with it. Researchers want to learn how the condition progresses over time in males and females. There are no new treatments given; it's purely about observing the disease.
United States · Germany · Israel - recruitingPHASE2
A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease
This study is looking at a new gene therapy called RP-A501 for boys and men with Danon Disease. It aims to check if the treatment is safe and helps improve their condition. It's a Phase 2 trial, meaning it's still being tested.
United States · Germany · Italy