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.
FDA Accepts sNDA for Higher Dosing Regimen of Nusinersen for Spinal Muscular Atrophy
January 23rd 2025In the phase 2/3 DEVOTE study, a higher dose of nusinersen met its primary end point at 6 months, achieving a statistically significant improvement in motor function among treatment-naïve symptomatic infants with SMA.
Expanding Knowledge on the Role of Neuropilin-1 in Chronic Pain: Nigel Bunnett, PhD, BSc
January 21st 2025The professor and chair of the Department of Molecular Pathobiology at NYU College of Dentistry discussed how a recently published preclinical study adds to the understanding of neuropilin-1 in chronic pain. [WATCH TIME: 3 minutes]
Early Immunotherapy May Be Crucial to Prevent Axonal Damage in CIDP, Study Suggests
Published: January 20th 2025 | Updated: February 3rd 2025A new study highlighted the importance of initiating immunotherapy promptly in chronic inflammatory demyelinating polyneuropathy to prevent axonal damage and disability progression.
NeuroVoices: Nigel Bunnett, PhD, BSc, on Identifying a Novel Therapeutic Target for Chronic Pain
January 15th 2025The professor and chair of the Department of Molecular Pathobiology at NYU College of Dentistry discussed groundbreaking research on chronic pain, focusing on the discovery of new mechanisms involving nerve growth factor and its receptors.
Discovering a New Receptor for Nerve Growth Factor: Nigel Bunnett, PhD, BSc
January 10th 2025The professor and chair of the Department of Molecular Pathobiology at NYU College of Dentistry provided a clinical overview of preclinical research identifying a new receptor for nerve growth factor that plays an important role in pain signaling. [WATCH TIME: 4 minutes]
FDA Clears IND for Trial Assessing Gene Therapy SGT-212 in Friedreich Ataxia
January 8th 2025SGT-212 delivers full-length frataxin via MRI-guided intradentate nucleus infusion and intravenous administration to target mitochondrial dysfunction in neurons and cardiomyocytes, addressing both neurologic and cardiac symptoms.
Expanding the Clinical Care of Friedreich Ataxia Through Omaveloxlone and SKYCLARYS PASS Registry
January 4th 2025David Lynch, MD, PhD, a professor of neurology at the University of Pennsylvania Perelman School of Medicine, discussed the significance of a number of recently presented analyses highlighting omaveloxlone, the first approved therapy for Friedreich ataxia.