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Danon disease
MONDO:0010281A lysosomal glycogen storage disease characterized by severe cardiomyopathy and variable degrees of muscle weakness, frequently associated with intellectual deficit.
Also known as: ANTOPOL disease, Danon disease, Danon disease, X-linked dominant, GSD due to LAMP-2 deficiency, LAMP2 lysosomal glycogen storage disease, glycogen storage disease type IIb, glycogenosis due to LAMP-2 deficiency, lysosomal glycogen storage disease caused by mutation in LAMP2
6 clinical trials for this condition and its sub-types, 5 tagged with Danon disease itself.
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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…
Phase 2 • Sponsor: Rocket Pharmaceuticals Inc. • Aim: Disease control
Last updated Jun 27, 2026 12:28 UTC
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Can a national registry unlock the genetic secrets of glycogen storage diseases in indian children?
Knowledge-focused Recruiting nowThis study aims to create a nationwide registry of Indian children with hepatic glycogen storage diseases (GSDs), a group of inherited metabolic disorders affecting the liver and muscles. By collecting genetic, clinical, and outcome data from 250 children, researchers hope to map…
Sponsor: Institute of Liver and Biliary Sciences, India • Aim: Knowledge-focused
Last updated Aug 07, 2026 00:00 UTC
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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…
Sponsor: Rocket Pharmaceuticals Inc. • Aim: Knowledge-focused
Last updated Jul 30, 2026 00:00 UTC
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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…
Sponsor: Chinese Academy of Medical Sciences, Fuwai Hospital • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:13 UTC
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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…
Sponsor: Duke University • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:08 UTC