Gene Therapy for Male Patients With Danon Disease Using AAV9.LAMP2B (RP-A501)
A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease
Danon Disease Natural History Study
The Natural History of Danon Disease
Natural History of Danon Disease
Healthcare Utilization and Economic Burden of Cardiomyopathy in China
Analysis of Clinical features and Echocardiographic / ML algorithms (includes rare cardiomyopathies)
Rare Glycogen Storage Diseases Natural History Study