Indication
Danon Disease
2 clinical trials
1 product
Product
RP-A501Clinical trial
A Clinical Study Evaluating a Recombinant Adeno-Associated Virus Serotype 9 (rAAV9) Capsid Containing the Human Lysosome-Associated Membrane Protein 2 Isoform B (LAMP2B) Transgene (RP-A501; AAV9.LAMP2B) in Male Patients With DDStatus: Active (not recruiting), Estimated PCD: 2025-03-24
Clinical trial
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 DiseaseStatus: Recruiting, Estimated PCD: 2025-09-01