DUCHENNE MUSCULAR DYSTROPHY
Clinical trials for DUCHENNE MUSCULAR DYSTROPHY explained in plain language.
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Heart drug trial aims to shield young duchenne patients from cardiac damage
Disease control CompletedThis study tested whether a blood pressure/heart rate medication called nebivolol could prevent heart muscle weakness in boys with Duchenne muscular dystrophy. Fifty-one boys aged 10-15 with Duchenne but normal heart function at the start took either nebivolol or a placebo pill d…
Matched conditions: DUCHENNE MUSCULAR DYSTROPHY
Phase: PHASE3 • Sponsor: Assistance Publique - Hôpitaux de Paris • Aim: Disease control
Last updated Apr 02, 2026 05:12 UTC
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Safety check for duchenne drug: can patients tolerate it Long-Term?
Disease control CompletedThis study aimed to check the long-term safety of a drug called ataluren in people with Duchenne muscular dystrophy caused by a specific 'nonsense' genetic mutation. It involved 270 participants who had already taken ataluren in a previous trial, or were siblings of those partici…
Matched conditions: DUCHENNE MUSCULAR DYSTROPHY
Phase: PHASE3 • Sponsor: PTC Therapeutics • Aim: Disease control
Last updated Apr 01, 2026 14:41 UTC
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New hope for boys with muscular dystrophy as major drug trial completes
Disease control CompletedThis large, completed study tested whether two drugs, casimersen and golodirsen, could help boys with a specific genetic form of Duchenne muscular dystrophy. For the first 96 weeks, some participants received the drug while others received a placebo, followed by a period where ev…
Matched conditions: DUCHENNE MUSCULAR DYSTROPHY
Phase: PHASE3 • Sponsor: Sarepta Therapeutics, Inc. • Aim: Disease control
Last updated Apr 01, 2026 14:41 UTC
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One-Shot gene therapy tested to fight devastating muscle disease in boys
Disease control CompletedThis early-stage trial tested a one-time gene therapy injection for boys with a specific genetic form of Duchenne muscular dystrophy. The goal was to see if the treatment was safe and could help their muscles produce a functional version of a crucial protein called dystrophin. Th…
Matched conditions: DUCHENNE MUSCULAR DYSTROPHY
Phase: PHASE1, PHASE2 • Sponsor: Megan Waldrop • Aim: Disease control
Last updated Mar 30, 2026 14:31 UTC
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New hope for boys with Muscle-Wasting disease: safer steroid shows promise
Disease control CompletedThis study tested vamorolone, a new type of steroid medication, in boys with Duchenne muscular dystrophy (DMD). It aimed to see if the drug was safe and how the body processed it, while also looking for early signs of benefit. The trial included 54 boys, aged 2-4 and 7-18, who to…
Matched conditions: DUCHENNE MUSCULAR DYSTROPHY
Phase: PHASE2 • Sponsor: Santhera Pharmaceuticals • Aim: Disease control
Last updated Mar 30, 2026 14:30 UTC
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Home-Based breathing exercises tested to help kids with muscle disease breathe easier
Symptom relief CompletedThis study tested whether a special breathing muscle training program, delivered remotely through tele-rehabilitation, could help children with Duchenne muscular dystrophy. The goal was to see if this training could improve their lung strength, walking ability, and overall qualit…
Matched conditions: DUCHENNE MUSCULAR DYSTROPHY
Phase: NA • Sponsor: Izmir Katip Celebi University • Aim: Symptom relief
Last updated Apr 02, 2026 05:12 UTC
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Home videos could transform how we track muscle disease
Knowledge-focused CompletedThis study aimed to test a new way of measuring movement in people with Duchenne muscular dystrophy (DMD) using home videos. It involved 150 participants who recorded videos of specific movements at home, which were then scored by physical therapists. The goal was to see if this …
Matched conditions: DUCHENNE MUSCULAR DYSTROPHY
Sponsor: The Emmes Company, LLC • Aim: Knowledge-focused
Last updated Mar 24, 2026 12:02 UTC