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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…

Translarna (ataluren) treatment delayed the loss of walking abilities by more than five years in boys with Duchenne muscular dystrophy (DMD) who carry nonsense mutations in their DMD gene, according to a study based on real-world data. The investigational therapy also slowed lung function decline by almost two…

Football and science seem to be disparate fields of play at first glance, but the nonprofit Uplifting Athletes is finding common ground by leveraging the popularity of college gridiron games to fund research for rare diseases. Its nearly two dozen chapters — representing college football teams across the nation…

BBP-418, an investigational disease-modifying medication that aims to improve muscle strength and function in people with limb-girdle muscular dystrophy type 2i (LGMD2i), has been given fast track designation by the U.S. Food and Drug Administration (FDA). This designation aims to facilitate and speed the development and regulatory review of…

A new viral vector that is better than conventional vectors — tools designed to deliver genetic material into cells — at targeting muscle cells may prove useful in developing a more effective gene therapy for muscular dystrophy (MD), according to researchers. In fact, this new vector “is more than…

Benitec Biopharma is taking steps to get the needed regulatory clearances to start clinical testing in Europe and the U.S. of BB-301, its experimental gene therapy for oculopharyngeal muscular dystrophy or OPMD, a type of the genetic disease that typically first affects the eyelids and the throat. The…

A team of researchers received a $3-million grant from the Chan Zuckerberg Initiative to create a comprehensive blueprint of healthy skeletal muscle cells — whose dysfunction is associated with several rare muscle diseases, such as muscle dystrophy — as children grow. Muscular dystrophy comprises a group of more…