DUCHENNE MUSCULAR DYSTROPHY
Clinical trials for DUCHENNE MUSCULAR DYSTROPHY explained in plain language.
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Experimental gene therapy targets DMD patients with antibody barriers
Disease control TerminatedThis study tested a gene therapy (delandistrogene moxeparvovec) combined with a drug called imlifidase to help patients with Duchenne muscular dystrophy who have antibodies that could block the therapy. The goal was to see if it was safe and could produce dystrophin protein. Only…
Matched conditions: DUCHENNE MUSCULAR DYSTROPHY
Phase: PHASE1 • Sponsor: Sarepta Therapeutics, Inc. • Aim: Disease control
Last updated May 17, 2026 08:04 UTC
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DMD drug safety study halted early after enrolling just 2 patients
Disease control TerminatedThis study looked at the safety of a drug called golodirsen in boys with Duchenne muscular dystrophy (DMD) who can no longer walk. The drug is given weekly through a vein and works by skipping a part of the genetic code to help produce a shorter but functional protein. Only 2 par…
Matched conditions: DUCHENNE MUSCULAR DYSTROPHY
Phase: PHASE4 • Sponsor: Rare Disease Research, LLC • Aim: Disease control
Last updated May 17, 2026 07:59 UTC
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Experimental DMD drug shows promise in small early trial
Disease control TerminatedThis study tested a new drug called PGN-EDO51 in 7 people with Duchenne muscular dystrophy whose genetic mutation can be fixed by skipping a part of their DNA called exon 51. The main goal was to see if the drug is safe and tolerable when given through a vein over several months,…
Matched conditions: DUCHENNE MUSCULAR DYSTROPHY
Phase: PHASE2 • Sponsor: PepGen Inc • Aim: Disease control
Last updated May 15, 2026 11:55 UTC
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Experimental gene therapy tested in duchenne boys with immune barriers – study stopped early
Disease control TerminatedThis early-stage study tested a gene therapy (delandistrogene moxeparvovec) in 3 boys with Duchenne muscular dystrophy who had pre-existing antibodies that could block the therapy. To overcome this, the boys first received a procedure called plasmapheresis to remove those antibod…
Matched conditions: DUCHENNE MUSCULAR DYSTROPHY
Phase: PHASE1 • Sponsor: Sarepta Therapeutics, Inc. • Aim: Disease control
Last updated May 11, 2026 20:46 UTC
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Gene therapy for duchenne MD shows promise but study halted early
Disease control TerminatedThis study tested a single dose of a gene therapy called PF-06939926 in 23 people with Duchenne muscular dystrophy (DMD), both those who could still walk and those who could not. The main goal was to check safety and side effects over one year. The trial was stopped early, but re…
Matched conditions: DUCHENNE MUSCULAR DYSTROPHY
Phase: PHASE1 • Sponsor: Pfizer • Aim: Disease control
Last updated May 07, 2026 18:43 UTC
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Gene therapy safety tracked for a decade in duchenne patients
Knowledge-focused TerminatedThis study checks the long-term safety and effects of an experimental gene therapy called fordadistrogene movaparvovec in people with Duchenne muscular dystrophy who already received it in an earlier study. Participants will be followed for 10 years with yearly visits. The goal i…
Matched conditions: DUCHENNE MUSCULAR DYSTROPHY
Phase: PHASE3 • Sponsor: Pfizer • Aim: Knowledge-focused
Last updated May 13, 2026 15:58 UTC