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
-
Decade-Long safety watch for groundbreaking duchenne gene therapy
Disease control TerminatedThis study follows boys with Duchenne muscular dystrophy for 10 years after they received an experimental gene therapy in previous Pfizer trials. The goal is to monitor long-term safety and see how well the treatment continues to work over time. Participants will have annual chec…
Matched conditions: DUCHENNE MUSCULAR DYSTROPHY
Phase: PHASE3 • Sponsor: Pfizer • Aim: Disease control
Last updated Apr 03, 2026 14:43 UTC
-
First human test of gene therapy for devastating muscle disease
Disease control TerminatedThis early-stage study tested the safety of a single dose of an experimental gene therapy, called PF-06939926, in boys with Duchenne muscular dystrophy (DMD). The main goal was to see if the treatment was safe and tolerable, while also checking if it helped produce the missing mu…
Matched conditions: DUCHENNE MUSCULAR DYSTROPHY
Phase: PHASE1 • Sponsor: Pfizer • Aim: Disease control
Last updated Apr 03, 2026 14:42 UTC
-
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 14:57 UTC
-
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
-
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