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

~Estimated

~$10 – $1,050

Rocket Pharmaceuticals Inc.NCT06214507

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

~Estimated

~$10 – $8,750

Rocket Pharmaceuticals Inc.NCT06092034

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

~Estimated

~$10 – $750

Duke UniversityNCT06795152