124: Therapeutic Updates and Progress in Treating Becker Muscular Dystrophy

NeurologyLive® Mind Moments®

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In this episode, Joanne Donovan, MD, PhD, chief medical officer at Edgewise Therapeutics, sat down to discuss the clinical program of EDG-5506, an investigational drug in development for Becker muscular dystrophy, and the recent progress in treating and understanding Becker muscular dystrophy. She spoke on the mechanism of action of the drug, also known as sevasemten, and why the company believes it can be therapeutically beneficial for this patient population. Furthermore, Donovan spoke on the previous failures in drug development for Becker, advances in biomarker detection, and how other trials paved the way for the company's phase 2 study and open label extension, dubbed CANYON (NCT05291091) and GRAND CANYON. Furthermore, she spoke on the ways the clinical community has tried to improve the quality of life for patients with Becker, focusing on approaches and tactics that curve cardiac issues commonly seen in this group.
Looking for more Neuromuscular discussion? Check out the NeurologyLive® neuromuscular clinical focus page.
Episode Breakdown:

• 1:15 – Overview of CANYON study and extension phase
• 3:50 – Mechanism of action of EDG-5506
• 6:30 – How previous trial experiences shaped CANYON
• 10:10 – Neurology News Minute
• 11:50 – Ongoing challenges with treating and testing drugs for Becker
• 15:25 – Safety profile of EDG-5506
• 16:45 – Ways to tackle cardiac issues in Becker

The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here:

• Digital Therapeutic CT-132 Meets Primary End Point in Phase 3 Study of Preventive Migraine

• FDA Supports Alpha-Synuclein Seed Amplification Assay Biomarker for Clinical Trials in Parkinson Disease

• FDA Approves Subcutaneous Formulation of Ocrelizumab for Relapsing and Progressive Multiple Sclerosis

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