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Microdystrophin gene therapy effectively maintained long-term heart function in a mouse model of severe Duchenne muscular dystrophy (DMD), a study has found. The treatment prevented scar formation and inflammation in heart tissue, and maintained normal heart function over 18 months. These findings support the ongoing clinical trials evaluating…

A rare neurological problem that does not appear to be directly related to AOC 1001 — the experimental treatment for myotonic dystrophy type 1 (DM1) being given patients in a Phase 1/2 clinical trial — may be the reason for a partial hold placed on the study,…

DYNE-251, an investigational therapy for Duchenne muscular dystrophy (DMD), has received orphan drug and rare pediatric disease designations from the U.S. Food and Drug Administration (FDA). Orphan drug status is meant to support the development of therapies for rare conditions affecting less than 200,000 people in the U.S. It…

Treatment with Translarna (ataluren) helps to slow the decline in motor function for people with Duchenne muscular dystrophy (DMD) caused by nonsense mutations, according to new clinical trial data and analyses presented this month at the Muscular Dystrophy Association’s MDA Clinical & Scientific Conference. The work was funded…

Note: This story was updated March 31, 2023, to clarify that the presentation was given at the Muscular Dystrophy Association’s MDA Clinical & Scientific Conference. Three boys with Duchenne muscular dystrophy (DMD) treated with the experimental cell therapy DT-DEC01 in a clinical trial continue to show improvements in motor…

Four boys with Duchenne muscular dystrophy (DMD) who were treated with the gene therapy candidate SRP-9001 (delandistrogene moxeparvovec) in a proof-of-concept clinical trial continue to show improvements in motor function four years after dosing. That’s according to new data discussed this month at the Muscular Dystrophy Association’s MDA Clinical…

People with Duchenne muscular dystrophy (DMD) who are treated with Exondys 51 (eteplirsen) live significantly longer than would be expected without the treatment, by a median of more than five years, according to a new analysis. The longest survival times were generally seen among patients who started on…

Researchers are planning to enroll a new group of patients in a natural history study to collect data about how Duchenne muscular dystrophy (DMD) progresses and affects a person’s life. Natural history studies don’t test an experimental therapy, but instead work to collect detailed information on how a disease…

In a bid to further understand and solve potential gene therapy safety concerns in Duchenne muscular dystrophy (DMD), the Muscular Dystrophy Association (MDA) has awarded two research grants totaling nearly $400,000. The funding will allow the awarded researchers and their teams to develop new strategies to prevent immune system…

A new gene editing technique was able to fix the mutation that most commonly causes type 2A limb-girdle muscular dystrophy (LGMD), a new study reports. “We show an effective and precise repair to correct the most frequent [LGMD type 2A] mutation … Our approach is a promising source for…