The NeurologyLive® neuromuscular disease clinical focus page offers updates and information related to the clinical care and management of individuals with neuromuscular diseases such as amyotrophic lateral sclerosis, Duchenne muscular dystrophy, cerebral palsy, and spinal muscular atrophy, as well as rare diseases. This page consists of news, podcasts, and videos about the latest FDA actions, clinical guideline updates, interviews with physicians, study and clinical trial findings, and more.

Latest News

RAINBOWFISH Study in NEJM Highlights Risdiplam’s Impact in Presymptomatic SMA, Importance of Newborn Screening
RAINBOWFISH Study in NEJM Highlights Risdiplam’s Impact in Presymptomatic SMA, Importance of Newborn Screening

August 20th 2025

Phase 3 study demonstrates risdiplam's effectiveness in treating presymptomatic spinal muscular atrophy, showing improved outcomes for infants identified through newborn screening.

FDA Denies Approval for Friedreich Ataxia Agent Vatiquinone, Citing More Efficacy Needed
FDA Denies Approval for Friedreich Ataxia Agent Vatiquinone, Citing More Efficacy Needed

August 19th 2025

The Complement Connection in Guillain-Barre and Chronic Inflammatory Demyelinating Polyneuropathy
The Complement Connection in Guillain-Barre and Chronic Inflammatory Demyelinating Polyneuropathy

August 19th 2025

Akshay Vaishnaw, MD, PhD  (Credit: LinkedIn)
Newly Published Phase 3 SAPPHIRE Study Highlight Therapeutic Potential of Apitegromab in Spinal Muscular Atrophy

August 18th 2025

This Week on NeurologyLive® — August 18, 2025
This Week on NeurologyLive® — August 18, 2025

August 18th 2025

Latest CME Events & Activities

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