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Danon Disease
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Danon Disease trials you may qualify forClinical trial pipeline · Data from ClinicalTrials.gov
See which Danon Disease trials you may qualify forThe 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 patients…
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 l…
What is this study about? This research is focused on improving the care for people with rare heart muscle diseases, known as rare cardiomyopathies. These are u…
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…
This is a multicenter, international, non-interventional, natural history study designed to collect longitudinal retrospective clinical information on patients…
This is a non-randomized open-label Phase 1 study to evaluate the safety and toxicity of gene therapy using a recombinant adeno-associated virus serotype 9 (AAV…
Danon disease (DD) is a rare, X-linked disorder associated with severe cardiomyopathy, and in many cases, skeletal myopathy, and cognitive impairment caused by…