Apply to trial NCT06092034

A few quick questions so the study team can decide if you might be a fit.

RecruitingPhase 2Genetic intervention

A Gene Therapy Study of RP-A501 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 lysosome-associated membrane protein 2 isoform B (LAMP2B) transgene, in male patients with Danon Disease.

How this works

Answer a few questions
About 5 to 10 minutes. Skip-friendly where possible.
We connect you with the study team
When a site picks up your application, we introduce you to their team directly — with you on the thread.
The team reaches out to schedule screening
Usually within a few business days, via the contact you give.

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A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease

How this works

Answer a few questions

We connect you with the study team

The team reaches out to schedule screening