DUCHENNE MUSCULAR DYSTROPHY
Clinical trials for DUCHENNE MUSCULAR DYSTROPHY explained in plain language.
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Experimental drug tested for boys with Muscle-Wasting disease
Disease control TerminatedThis study tested an experimental drug called PGN-EDO51 in boys with Duchenne muscular dystrophy who have a specific genetic mutation. The main goal was to check if the drug is safe and how the body processes it when given through an IV over time. Researchers also measured whethe…
Matched conditions: DUCHENNE MUSCULAR DYSTROPHY
Phase: PHASE2 • Sponsor: PepGen Inc • Aim: Disease control
Last updated Apr 02, 2026 02:12 UTC
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First human test of gene therapy for devastating muscle disease
Disease control TerminatedThis was an early safety study by Pfizer for a new gene therapy, PF-06939926, for Duchenne muscular dystrophy (DMD). It tested a single intravenous dose in a small group of boys, both walking and non-walking, to see if it was safe and tolerable. The study also looked for early si…
Matched conditions: DUCHENNE MUSCULAR DYSTROPHY
Phase: PHASE1 • Sponsor: Pfizer • Aim: Disease control
Last updated Mar 31, 2026 12:11 UTC
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Gene therapy trial halted for boys with rare muscle disease
Disease control TerminatedThis study tested a two-step treatment for Duchenne muscular dystrophy (DMD). First, a drug (imlifidase) was given to temporarily lower antibodies that might block the gene therapy. Then, the gene therapy (SRP-9001) was given to try to help muscles produce a needed protein. The s…
Matched conditions: DUCHENNE MUSCULAR DYSTROPHY
Phase: PHASE1 • Sponsor: Sarepta Therapeutics, Inc. • Aim: Disease control
Last updated Mar 30, 2026 14:31 UTC
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10-Year watch: tracking boys after groundbreaking gene therapy
Disease control TerminatedThis study follows boys with Duchenne muscular dystrophy for 10 years after they received an experimental gene therapy called fordadistrogene movaparvovec in earlier Pfizer trials. The main goal is to monitor long-term safety and see how well the treatment continues to work over …
Matched conditions: DUCHENNE MUSCULAR DYSTROPHY
Phase: PHASE3 • Sponsor: Pfizer • Aim: Disease control
Last updated Mar 30, 2026 14:29 UTC
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Blood filtering paves way for gene therapy in boys with rare muscle disease
Disease control TerminatedThis early-stage study tested a one-time gene therapy for Duchenne muscular dystrophy (DMD) in a very specific group: three boys who already had antibodies that might block the treatment. To try and get around this, doctors first used a blood-filtering procedure called plasmapher…
Matched conditions: DUCHENNE MUSCULAR DYSTROPHY
Phase: PHASE1 • Sponsor: Sarepta Therapeutics, Inc. • Aim: Disease control
Last updated Mar 27, 2026 12:38 UTC