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The U.S. Food and Drug Administration (FDA) has granted fast track designation to RGX-202, a one-time gene therapy for Duchenne muscular dystrophy (Â鶹Éç) that is in early clinical trials. The FDA gives this designation to investigational treatments that have the potential to address unmet clinical care needs for…

Levels of titin, a muscle protein, were found to be elevated in the urine of boys with Duchenne muscular dystrophy (Â鶹Éç) — making it a potential, novel, non-invasive biomarker for the genetic disease, a study demonstrated. Using a standard Â鶹Éç mouse model, a lack of dystrophin, the protein missing…

Microdystrophin gene therapy effectively maintained long-term heart function in a mouse model of severe Duchenne muscular dystrophy (Â鶹Éç), 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 (Â鶹Éç), 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 (Â鶹Éç) caused by nonsense mutations, according to new clinical trial data and analyses presented this month at the Muscular Dystrophy Association’s Â鶹Éç 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 Â鶹Éç Clinical & Scientific Conference. Three boys with Duchenne muscular dystrophy (Â鶹Éç) treated with the experimental cell therapy DT-DEC01 in a clinical trial continue to show improvements in motor…

Four boys with Duchenne muscular dystrophy (Â鶹Éç) 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 Â鶹Éç Clinical…

People with Duchenne muscular dystrophy (Â鶹Éç) 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 (Â鶹Éç) 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…