News

The Newborn Screening Pilot program run in New York by Parent Project Muscular Dystrophy (PMDD) is now complete, having screened more than 36,000 babies across the state for a muscular dystrophy over the last two years. Significantly high levels of creatine kinase, an enzyme released by damaged muscle cells that can indicate…

Flying can be an ordeal for many people with disabilities who rely on wheelchairs to get from point A to point B. Sometimes disabled travelers get to their destination only to realize their wheelchair or scooter is broken or missing. In 2019, the year after airlines were required to release…

The COVID-19 pandemic has had a substantial impact on the mental health and quality of life of people affected by muscular dystrophy (MD). One big underlying issue for the families of those with Duchenne and Becker muscular dystrophies is that the pandemic has caused many patients to…

The U.S. Food and Drug Administration (FDA) has granted fast track designation to AOC 1001, an investigational treatment for myotonic dystrophy type 1, known as DM1, a late-developing muscle disorder. The designation will enable AOC 1001’s developer, Avidity Biosciences, to have more frequent interactions with the FDA throughout…

New data across clinical trials of SRP-9001 show the investigative gene therapy induces sustained functional improvements in people with Duchenne muscular dystrophy (DMD), and does so with good tolerability, Sarepta Therapeutics, its developer, announced. The company, in presenting these data, also offered more details about the recently launched and pivotal…

Tamoxifen, commonly used to treat breast cancer, failed to meet its primary goal — delaying disease progression — in a Phase 3 study evaluating its use in boys with Duchenne muscular dystrophy (DMD), preliminary data show. Boys treated with tamoxifen and those given a placebo over 48 weeks in the…

Note: This story was updated Oct. 8, 2021, to clarify that Sarepta did not confirm previously announced plans to enroll 75 boys between the ages of 4 and 7 in the EMBARK trial in its latest announcement. Sarepta Therapeutics and Roche have announced the launch of EMBARK,…

Brain abnormalities in people with adult-onset myotonic dystrophy type 1 (DM1) are significantly associated with cognitive impairment, apathy, and daytime sleepiness, a study shows. Notably, no such link was found for depression and anxiety, suggesting that these commonly reported symptoms are instead secondary manifestations of living and coping with…

Treatment with SGT-001 — Solid Biosciences’ gene therapy candidate for Duchenne muscular dystrophy (DMD) — improves lung function, according to data from the first six patients enrolled in the ongoing IGNITE DMD clinical trial. The improvements, seen one year after a single infusion of the SGT-001 gene therapy into…

Treatment with the investigational cell therapy CAP-1002 significantly improved arm and heart function in boys and young men at advanced stages of Duchenne muscular dystrophy (DMD), according to final data from the HOPE-2 clinical trial. The Phase 2 trial also “met various skeletal and cardiac endpoints [goals] suggesting clinically…