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Phase 2
Gene Therapy for Danon Disease: A Phase 2 Study Evaluating the Efficacy and Safety of Intravenously Administered Adeno-Associated Virus Serotype 9 (rAAV9) Vector Containing the Human LAMP2 Isoform B Transgene (RP-A501; AAV9.LAMP2B) in Male Patients With Danon Disease
This is a single arm Phase 2 trial to evaluate the efficacy and safety of RP-A501, a recombinant adeno-associated virus serotype 9 (AAV9) containing the human …
Danon Disease
Rocket Pharmaceuticals Inc.NCT06092034
An Observational Study of Genetic Cardiomyopathy, Danon Disease
The goal of this international observational study is to learn about the natural history of Danon disease in male patients (≥8 years of age) and female patient…
Danon Disease
Rocket Pharmaceuticals Inc.NCT06214507
Rare Glycogen Storage Diseases Natural History Study
The purpose of this study is to collect and study key medical data about several ultra-rare GSDs (Glycogen Storage Diseases) including, but not limited to, GSD…
Glycogen Storage DiseaseGSD Type 0AGSD Type 0B+12 more
Duke UniversityNCT06795152