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The U.S. Food and Drug Administration (FDA) has granted orphan drug status to NS-051/NCNP-04, an exon-skipping therapy designed to treat certain people with Duchenne muscular dystrophy (DMD). The FDA gives this designation to experimental treatments designed to improve care for rare disorders, specifically conditions affecting fewer than 200,000 people…

A Phase 1/2 clinical trial testing SAR446268, an experimental gene therapy for myotonic dystrophy type 1 (DM1), is enrolling patients. The trial, dubbed BrAAVe (NCT06844214), is expected to enroll approximately 32 DM1 patients aged 10-50. The study is actively recruiting participants at sites in Florida, New York,…

A combined regimen of testosterone and growth hormone led to significant improvements in muscle mass, strength, and mobility in men with facioscapulohumeral muscular dystrophy (FSHD) in a clinical trial. The Phase 1/2 STARFISH trial (NCT03123913) evaluated the combination therapy’s safety, tolerability, and potential efficacy in men with FSHD.

Long-term treatment with Duvyzat (givinostat) delays the loss of key motor skills in people with Duchenne muscular dystrophy (DMD), published results from an open-label extension study show. “The sustained benefit observed across functional outcomes reinforces the potential of Duvyzat to meaningfully alter the course of the disease,” Scott…

Note: This story was updated Sept. 10, 2025, to correct the secondary headline from Columbia University to the University of Iowa. Columbia University awarded the 2025 Louisa Gross Horwitz Prize to three scientists whose research has helped lay the groundwork to develop new treatments for Duchenne muscular dystrophy (DMD). This…

Researchers have developed a way to enhance the efficacy of exon-skipping therapies for Duchenne muscular dystrophy (DMD), and the approach showed promise in a mouse model of the disease. The study, “A Combinatorial Oligonucleotide Therapy to Improve Dystrophin Restoration and Dystrophin-Deficient Muscle Health,” was published in Molecular Therapy…

Duchenne muscular dystrophy (DMD) treatment Exondys 51 (eteplirsen) may help protect the heart as well as muscles used for movement, a study found. The treatment slowed heart function decline, raising the possibility it could lower the risk of cardiomyopathy, a serious condition that can lead to heart failure.

Across the U.S., the neuromuscular disease community is preparing to mark Muscular Dystrophy Awareness Month this September, with a series of events and activities aimed at spotlighting muscular dystrophy (MD) and other related conditions. These efforts, organizers say, also seek to raise vital funds to support all individuals in…

Levels of certain proteins implicated in immune response and the body’s process to stop bleeding differ between Becker muscular dystrophy (BMD) and Duchenne muscular dystrophy (DMD), according to a study Also, blood levels of these proteins correlated with the degree of motor function in BMD participants, but not…

Researchers have developed a detailed molecular model for the structure of type VI collagen and identified where genetic mutations linked to certain forms of muscular dystrophy (MD) might influence the protein. Findings from their study could ultimately help scientists better understand the mechanisms underlying types of MD that affect…