DANON DISEASE
Clinical trials for DANON DISEASE explained in plain language.
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One-Time infusion aims to fix faulty heart gene
Disease control Recruiting nowThis study is testing a one-time gene therapy infusion for males aged 8 and older with Danon Disease, a rare genetic condition that damages the heart. The goal is to see if delivering a corrected gene can improve heart health and slow disease progression. Researchers will measure…
Matched conditions: DANON DISEASE
Phase: PHASE2 • Sponsor: Rocket Pharmaceuticals Inc. • Aim: Disease control
Last updated Mar 27, 2026 12:38 UTC
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Scientists gather clues about Ultra-Rare metabolic disorders
Knowledge-focused Recruiting nowThis study aims to collect medical information from people with several ultra-rare glycogen storage diseases (GSDs) to better understand how these conditions progress over time. Researchers will review medical records from about 200 participants to create a detailed database of s…
Matched conditions: DANON DISEASE
Sponsor: Duke University • Aim: Knowledge-focused
Last updated Mar 27, 2026 12:40 UTC
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New heart scans aim to solve diagnostic mysteries of rare cardiac conditions
Knowledge-focused Recruiting nowThis study is testing whether advanced heart imaging techniques can improve how doctors diagnose and predict risks for rare heart muscle diseases. Researchers will use special heart scans to create detailed pictures of participants' hearts, looking for patterns that might help id…
Matched conditions: DANON DISEASE
Sponsor: Chinese Academy of Medical Sciences, Fuwai Hospital • Aim: Knowledge-focused
Last updated Mar 27, 2026 12:37 UTC
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Scientists track rare genetic heart disease to find better treatments
Knowledge-focused Recruiting nowThis study aims to understand how Danon disease, a rare and severe genetic heart condition, progresses over time in patients. Researchers will collect medical information from about 60 male and female patients, both from their past records and by following them forward. The goal …
Matched conditions: DANON DISEASE
Sponsor: Rocket Pharmaceuticals Inc. • Aim: Knowledge-focused
Last updated Mar 16, 2026 15:25 UTC