Episode 80: Advancing Treatment for Rare Neuromuscular Disorders

Sarah Boyce

Episode 80 of the NeurologyLive^® Mind Moments^® podcast is now live! Scroll down to listen or click here to subscribe on your favorite streaming service.

The Mind Moments^® podcast features exclusive interviews with leaders in the field discussing the latest research and disease management strategies across the breadth of neurology, including epilepsy, multiple sclerosis, Parkinson disease, dementia, sleep disorders, and more.

Episode 80, "Advancing Treatment for Rare Neuromuscular Disorders," features an exclusive interview 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.

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.

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

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

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