80: Advancing Treatment for Rare Neuromuscular Disorders

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In this episode, we spoke with Sarah Boyce, president and CEO of Avidity Biosciences, who shared insight into the company's ongoing work with its antibody oligonucleotide conjugates (AOC) platform and the focus on myotonic dystrophy type 1 (DM1), Duchenne muscular dystrophy (DMD), and facioscapulohumeral muscular dystrophy (FSHD) treatments, as well as the challenges with developing successful RNA therapies, and more. Looking for more neuromuscular disorder discussion? Check out the NeurologyLive® neuromuscular clinical focus page: neurologylive.com/clinical/neuromuscular Episode Breakdown: 1:20 – Current limitations with RNA therapies 4:50 – The decision to focus on rare genetic muscle diseases 7:45 – The patient needs in DM1, FSHD, and DMD 9:05 – Neurology News Minute 12:25 – Other clinical areas of focus and collaboration for Avidity 14:10 – Upcoming data presentations planned for 2023 in DMD and myotonic dystrophy 16:15 – The benefits of collaboration in the rare disease community 18:15 – Looking ahead to the future of therapies in rare muscular disease This episode is brought to you by the Giants of Multiple Sclerosis®. This premier neuroscience award program celebrates pioneers, innovators, and future generations of leaders for their remarkable achievements in Multiple Sclerosis. Nominations close January 31, 2023! Nominate: neurologylive.com/Giants-of-MS 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: Anti-CD20 Therapy Ublituximab Gains FDA Approval for Relapsing Multiple Sclerosis FDA Clears First Stroke-Specific Radial Access Platform for Mechanical Thrombectomy FDA Designates Myasthenia Gravis Agent Rozanolixizumab Application for Priority Review FDA Approves Eisai’s Lecanemab for the Treatment for Alzheimer Disease FDA Accepts sBLA for Revance’s Cervical Dystonia Treatment DaxibotulinumtoxinA Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.