Danon Disease clinical trials at University of California Health

2 research studies open to eligible people

Showing trials for

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

    open to eligible males ages 8 years and up

    This is a single arm Phase 2 trial to evaluate the efficacy and safety of RP-A501, a recombinant adeno-associated virus serotype 9 (rAAV9) vector containing the human lysosome-associated membrane protein 2 isoform B (LAMP2B) transgene, in male patients with Danon disease.

    at UCSD

  • Danon Disease Natural History Study

    open to eligible people ages 8 years and up

    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 patients (8 to 50 years of age).

    at UCSD

Our lead scientists for Danon Disease research studies include .