DUCHENNE MUSCULAR DYSTROPHY
Clinical trials for DUCHENNE MUSCULAR DYSTROPHY explained in plain language.
Never miss a new study
Get alerted when new DUCHENNE MUSCULAR DYSTROPHY trials appear
Sign up with your email to follow new studies for DUCHENNE MUSCULAR DYSTROPHY, keep track of the ones that matter, and come back to a personal dashboard instead of checking manually.
Genom att skicka in godkänner du våra Användarvillkor
-
DMD drug safety trial ends early with just 2 participants
Disease control TerminatedThis study looked at the safety of a drug called golodirsen in boys with Duchenne muscular dystrophy who can no longer walk. The drug is given as a weekly IV infusion. The study was stopped early and only included 2 participants, so results are very limited.
Matched conditions: DUCHENNE MUSCULAR DYSTROPHY
Phase: PHASE4 • Sponsor: Rare Disease Research, LLC • Aim: Disease control
Last updated May 26, 2026 04:43 UTC
-
Gene therapy watch: 10-Year safety check for duchenne patients
Disease control TerminatedThis study checks the long-term safety and effects of an experimental gene therapy called fordadistrogene movaparvovec in people with Duchenne muscular dystrophy who received it in a previous Pfizer study. Participants will be followed for up to 10 years with annual visits. The g…
Matched conditions: DUCHENNE MUSCULAR DYSTROPHY
Phase: PHASE3 • Sponsor: Pfizer • Aim: Disease control
Last updated May 26, 2026 04:36 UTC
-
Experimental gene therapy tested in DMD patients with antibodies
Disease control TerminatedThis study tested a gene therapy called delandistrogene moxeparvovec in 3 boys with Duchenne muscular dystrophy who had antibodies that could block the treatment. Before receiving the therapy, participants underwent a procedure called plasmapheresis to remove those antibodies. Th…
Matched conditions: DUCHENNE MUSCULAR DYSTROPHY
Phase: PHASE1 • Sponsor: Sarepta Therapeutics, Inc. • Aim: Disease control
Last updated May 22, 2026 14:01 UTC
-
Experimental gene therapy for DMD hits antibody barrier
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. Only 5 participants were planned, and the study was terminated early. The goa…
Matched conditions: DUCHENNE MUSCULAR DYSTROPHY
Phase: PHASE1 • Sponsor: Sarepta Therapeutics, Inc. • Aim: Disease control
Last updated May 22, 2026 13:54 UTC
-
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
-
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