DANON DISEASE
Clinical trials for DANON DISEASE explained in plain language.
Never miss a new study
Get alerted when new DANON DISEASE trials appear
Sign up with your email to follow new studies for DANON DISEASE, keep track of the ones that matter, and come back to a personal dashboard instead of checking manually.
By submitting, you agree to our Terms of use
-
Gene therapy breakthrough offers hope for rare heart disease
Disease control Recruiting nowThis study tests a gene therapy called RP-A501 in 14 males 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, aiming to improve heart muscle function and reduce thickening. Part…
Matched conditions: DANON DISEASE
Phase: PHASE2 • Sponsor: Rocket Pharmaceuticals Inc. • Aim: Disease control
Last updated Jun 27, 2026 12:28 UTC
-
New study tracks rare heart disease to pave way for future treatments
Knowledge-focused Recruiting nowThis study follows 60 people with Danon disease, a rare genetic heart condition, to learn how it progresses over time. Researchers will monitor heart function and symptoms in males aged 8 and older and females aged 8 to 50. The goal is to gather natural history data that could su…
Matched conditions: DANON DISEASE
Sponsor: Rocket Pharmaceuticals Inc. • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:28 UTC
-
New scan techniques aim to solve rare heart disease mysteries
Knowledge-focused Recruiting nowThis study is testing advanced heart MRI scans to better diagnose and predict risks for people with rare heart muscle diseases. Researchers will scan 1000 participants to see if these new imaging methods can identify conditions like Fabry disease and cardiac amyloidosis more accu…
Matched conditions: DANON DISEASE
Sponsor: Chinese Academy of Medical Sciences, Fuwai Hospital • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:13 UTC
-
Scientists launch Largest-Ever study of Ultra-Rare GSDs
Knowledge-focused Recruiting nowThis study tracks 200 people with ultra-rare glycogen storage diseases (GSDs) like types 0a, 0b, VII, X, XII, XIII, XV, PRKAG2 syndrome, and Danon disease. Researchers will collect medical records and other data to see how these conditions progress over time. No treatments or dru…
Matched conditions: DANON DISEASE
Sponsor: Duke University • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:08 UTC