Danon Disease clinical trials at UCSD
2 research studies open to eligible people
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 (AAV9) containing the human lysosome-associated membrane protein 2 isoform B (LAMP2B) transgene, in male patients with Danon Disease.
La Jolla, California and other locations
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).
La Jolla, California and other locations
Our lead scientists for Danon Disease research studies include Kimberly Hong, MD Barry Greenberg, MD.
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