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
-
Experimental 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 weakens the heart. The therapy uses a harmless virus to deliver a working copy of the LAMP2 gene, aiming to improve heart muscle function and reduce thick…
Matched conditions: DANON DISEASE
Phase: PHASE2 • Sponsor: Rocket Pharmaceuticals Inc. • Aim: Disease control
Last updated May 15, 2026 11:57 UTC
-
Researchers track rare heart disease to unlock clues for future therapies
Knowledge-focused Recruiting nowThis study observes 60 people with Danon disease, a rare genetic heart condition, to see how it changes over time. Participants include males 8 and older and females 8 to 50. The goal is to better understand the disease, not to test a treatment.
Matched conditions: DANON DISEASE
Sponsor: Rocket Pharmaceuticals Inc. • Aim: Knowledge-focused
Last updated May 17, 2026 04:17 UTC
-
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 17, 2026 04:17 UTC
-
Scientists launch study to track rare GSDs
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 better unde…
Matched conditions: DANON DISEASE
Sponsor: Duke University • Aim: Knowledge-focused
Last updated May 15, 2026 11:56 UTC