DANON DISEASE
Clinical trials for DANON DISEASE explained in plain language.
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Gene therapy offers hope for rare heart disease
Disease control Recruiting nowThis study tests a gene therapy called RP-A501 in 14 males aged 8 and older with Danon disease, a rare genetic condition that causes heart problems. The treatment uses a harmless virus to deliver a working copy of the LAMP2 gene directly into the body. The goal is to improve hear…
Matched conditions: DANON DISEASE
Phase: PHASE2 • Sponsor: Rocket Pharmaceuticals Inc. • Aim: Disease control
Last updated May 26, 2026 04:29 UTC
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Rare disease data hunt: no cure, but crucial insights ahead
Knowledge-focused Recruiting nowThis study aims to collect and analyze medical information from 200 people with ultra-rare glycogen storage diseases (GSDs), including types 0a, 0b, VII, X, XII, XIII, XV, PRKAG2 syndrome, and Danon disease. Participants must have a confirmed diagnosis. The goal is to track how t…
Matched conditions: DANON DISEASE
Sponsor: Duke University • Aim: Knowledge-focused
Last updated May 26, 2026 04:39 UTC
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Researchers track rare heart disease to unlock its secrets
Knowledge-focused Recruiting nowThis study follows 60 people with Danon disease, a rare genetic heart condition, to see how it progresses. Participants include males 8 and older and females 8 to 50. Researchers will measure heart size, blood markers, and quality of life to better understand the disease and help…
Matched conditions: DANON DISEASE
Sponsor: Rocket Pharmaceuticals Inc. • Aim: Knowledge-focused
Last updated May 26, 2026 04:35 UTC
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New heart scan techniques could unlock secrets of rare heart diseases
Knowledge-focused Recruiting nowThis study aims to improve how doctors diagnose and predict the course of rare heart muscle diseases (cardiomyopathies) using advanced MRI scans. Researchers will enroll 1000 people with suspected rare cardiomyopathies to test new imaging methods. The goal is to provide clearer, …
Matched conditions: DANON DISEASE
Sponsor: Chinese Academy of Medical Sciences, Fuwai Hospital • Aim: Knowledge-focused
Last updated May 18, 2026 12:00 UTC