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.
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.
European Commission Approves Efgartigimod Subcutaneous Injection for Forms of CIDP
June 24th 2025Efgartigimod alfa SC is now approved in Europe for patients with chronic inflammatory demyelinating polyneuropathy, following a positive recommendation from the Committee for Medicinal Products for Human Use.
Strengthening Duchenne Care Through Clinical Collaboration and Community Focus: Brenda L. Wong, MD
June 15th 2025The director of the DMD Program at UMass Chan Medical School shared her experience at the 2025 CureDuchenne FUTURES National Conference, held May 22-25, in San Antonio, Texas. [WATCH TIME: 5 minutes]
Insights on NervGen Pharma’s Positive Phase 1b/2a Data for NVG-291 in Spinal Cord Injury
June 14th 2025Daniel Mikol, MD, PhD, chief medical officer at NervGen Pharma, provided clinical perspective on recently announced positive findings from a phase 1b/2a trial testing NVG-291 as a treatment for spinal cord injury.
Emerging Considerations in Peripheral Nerve Injury and Surgical Intervention: Kevin Swong, MD
June 12th 2025The neurosurgeon at Northwestern Medicine discussed diagnostic approaches, evolving treatment strategies, and the importance of timely referral in managing peripheral nerve injuries. [WATCH TIME: 3 minutes]
National RLS Registry Provides Long-Term Insights Into Opioid Use for Restless Legs Syndrome
June 12th 2025New 5-year findings from the National RLS Opioid Registry presented at SLEEP 2025 highlight the long-term stability and dose trends of low-dose opioid therapy in patients with restless legs syndrome.
Rationale Behind NVG-2089 as New Therapy for CIDP: Pamela Conley; Alan Glicklich, MD
June 12th 2025Members from Nuvig Therapeutics discussed how NVG-2089 could improve the CIDP treatment landscape by offering IVIG-like efficacy with a better safety and administration profile. [WATCH TIME: 3 minutes]
Reactivating Muscle Stem Cells in DMD: The Science Behind SAT-3247
June 5th 2025Phil Lambert, PhD, chief scientific officer at Satellos Bioscience, discusses the rationale and early clinical progress of SAT-3247, a novel regenerative therapy targeting muscle stem cells in Duchenne muscular dystrophy.