DUCHENNE MUSCULAR DYSTROPHY
Clinical trials for DUCHENNE MUSCULAR DYSTROPHY explained in plain language.
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DMD drug tested in wheelchair users – but trial stops early
Disease control TerminatedThis study tested the safety of golodirsen (Vyondys 53) in boys and men with Duchenne muscular dystrophy who can no longer walk. Only 2 people took part before the trial was stopped early. Participants received weekly IV infusions for up to 96 weeks, with extra follow-up. The goa…
Matched conditions: DUCHENNE MUSCULAR DYSTROPHY
Phase: PHASE4 • Sponsor: Rare Disease Research, LLC • Aim: Disease control
Last updated Jun 27, 2026 12:03 UTC
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New drug trial for duchenne MD halted early – what we know
Disease control TerminatedThis phase 2 study tested a drug called PGN-EDO51 in 7 people with Duchenne muscular dystrophy whose genetic mutation can be corrected by skipping exon 51. The drug was given by IV infusion to see if it is safe and tolerable. The trial was terminated, so results are limited.
Matched conditions: DUCHENNE MUSCULAR DYSTROPHY
Phase: PHASE2 • Sponsor: PepGen Inc • Aim: Disease control
Last updated Jun 27, 2026 08:01 UTC
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Experimental gene therapy for DMD hits antibody barrier – study halted
Disease control TerminatedThis study tested a gene therapy (delandistrogene moxeparvovec) combined with a drug called imlifidase to see if it could safely deliver the therapy to boys with Duchenne muscular dystrophy who had antibodies that might block the treatment. Only 5 participants were planned, but t…
Matched conditions: DUCHENNE MUSCULAR DYSTROPHY
Phase: PHASE1 • Sponsor: Sarepta Therapeutics, Inc. • Aim: Disease control
Last updated Jun 27, 2026 07:59 UTC
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Gene therapy for duchenne muscular dystrophy under Long-Term watch
Disease control TerminatedThis study follows 7 people with Duchenne muscular dystrophy who previously received an experimental gene therapy called fordadistrogene movaparvovec. Researchers will monitor them for 10 years to check for side effects and see if the treatment continues to help with movement. Th…
Matched conditions: DUCHENNE MUSCULAR DYSTROPHY
Phase: PHASE3 • Sponsor: Pfizer • Aim: Disease control
Last updated Jun 27, 2026 07:55 UTC
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Gene therapy trial for duchenne MD halted early – what we know
Disease control TerminatedThis early-stage trial tested a single infusion of gene therapy (PF-06939926) in 23 people with Duchenne muscular dystrophy, both those who could still walk and those who could not. The main goal was to check safety and tolerability, while also measuring dystrophin protein levels…
Matched conditions: DUCHENNE MUSCULAR DYSTROPHY
Phase: PHASE1 • Sponsor: Pfizer • Aim: Disease control
Last updated Jun 26, 2026 17:12 UTC
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Can plasma exchange clear the way for gene therapy in duchenne?
Disease control TerminatedThis early study tested whether a blood-cleaning procedure called plasmapheresis could allow boys with Duchenne muscular dystrophy who have antibodies against the gene therapy carrier to still receive the treatment. Only 3 boys were enrolled before the study was stopped early. Th…
Matched conditions: DUCHENNE MUSCULAR DYSTROPHY
Phase: PHASE1 • Sponsor: Sarepta Therapeutics, Inc. • Aim: Disease control
Last updated Jun 26, 2026 14:20 UTC