Spotlight on Becker Muscular Dystrophy: Understanding the Lived Experience of Becker and Clinical Advancements with a Novel Agent

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This symposium was held at the Muscular Dystrophy Association (MDA) Clinical & Scientific Conference 2025, in Dallas, Texas, USA. Speakers gave an overview of the patient experience and natural history of Becker muscular dystrophy (Becker) and presented the latest Phase I/II clinical data for the novel investigational agent, sevasemten. Becker is a serious, rare, neuromuscular disorder that produces irreversible muscle loss and progressive impairments in mobility and cardiac function, amongst other physical and emotional effects. However, there are currently no approved treatments for Becker. In a powerful start to the symposium, Michael Voto, New Jersey, USA, a patient advocate living with Becker, provided insight into the lived experience of Becker and, together with Abby Bronson, Vice-President of Patient Advocacy, Edgewise Therapeutics, Colorado, USA, highlighted the importance of the patient voice to improve awareness, and create consistent standard-of-care in Becker. Craig McDonald, Director of the MDA Neuromuscular Diseases Clinics, University of California Davis, USA, explained how understanding of the natural history of Becker can better direct the use of functional measures such as the North Star Ambulatory Assessment (NSAA), as a meaningful outcome in Becker clinical studies. McDonald also outlined clinical advancements with the fast myofibre (Type II) myosin inhibitor, sevasemten (formerly EDG-5506), including new data from CANYON, the largest randomised, controlled clinical trial in Becker to date. CANYON met its primary endpoint demonstrating significant decreases in biomarkers of muscle damage with sevasemten treatment, as well as stabilised ambulatory function (NSAA) trending to improvement, which is a clinically meaningful outcome for patients.