Neuromuscular

Long-term data confirms givinostat's safety and efficacy in delaying Duchenne muscular dystrophy progression, offering hope for patients and families.

Amylyx Pharmaceuticals discontinued its AMX0035 program for progressive supranuclear palsy after a phase 2b trial failed to show efficacy.

MediciNova's COMBAT-ALS trial reaches full enrollment, exploring ibudilast's potential to treat ALS and improve patient outcomes.

The neurophysiologist at Allegheny Health Network shared his clinical experience with tofersen in patients with SOD-1 ALS, while emphasizing the critical role of accessible genetic testing for patient identification. [WATCH TIME: 3 minutes]

Tracey Dawson, PhD, senior vice president of U.S. Therapeutic Area Head of Neuroscience at Novartis, expanded on the recently launched SMAshing My Limits campaign, a community-driven project aimed to address needs of developing teens with SMA.

The vice chair of education for the department of neurology at Mass General outlined her AUPN 2025 session on supporting clinician educators through recognition, career development, and institutional frameworks for advancement. [WATCH TIME: 4 minutes]

Serum NPTX2 emerges as a novel biomarker in ALS, with combined NPTX2 and NfL levels improving survival prediction and disease monitoring.

Regeneron's cemdisiran shows promising results in treating generalized myasthenia gravis, paving the way for a potential new drug application in 2026.

The neurologist at Maimonides Medical Center and SUNY Downstate shared how interactive simulations give neurology trainees the chance to practice skills, gain feedback, and move beyond passive learning. [WATCH TIME: 3 minutes]

The FDA has accepted the IND for COYA 302, a Treg-targeting combination therapy for ALS, clearing the way for a pivotal phase 2 multicenter trial.

Efgartigimod shows promise in treating seronegative myasthenia gravis, offering hope for patients with limited options and paving the way for new therapies.

Here's some of what is coming soon to NeurologyLive® this week.

Neurology News Network. for the week ending August 23, 2025. [WATCH TIME: 4 minutes]

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

PTC Therapeutics faces FDA setback for vatiquinone, a potential Friedreich ataxia treatment, requiring further studies to demonstrate efficacy.

Explore the role of the complement system in autoimmune neuropathies like GBS and CIDP, and discover innovative therapeutic strategies targeting complement pathways.

Scholar Rock’s phase 3 SAPPHIRE trial of apitegromab revealed that the treatment led to improved motor function in patients with spinal muscular atrophy over a 52-week period.

Here's some of what is coming soon to NeurologyLive® this week.

Neurology News Network. for the week ending August 16, 2025. [WATCH TIME: 4 minutes]

Novartis revealed promising phase 3 trial results for ianalumab, a potential first treatment for Sjögren’s disease, showing significant disease activity reduction.

Catch up on any of the neurology news headlines you may have missed over the course of July 2025, compiled all into 1 place by the NeurologyLive® team.

Here's some of what is coming soon to NeurologyLive® this week.

Daniel Freedman, PhD, of MIT, gave clinical commentary on how Openwater’s Open-LIFU enables new research into conscious perception and potential clinical applications for conditions like pain and depression.

Vertex Pharmaceuticals halts VX-993 development for acute pain after phase 2 trial shows no significant efficacy compared with placebo.

The neurologist at Duke Health provided insights on how chronic demyelinating inflammatory polyneuropathy treatment is evolving toward biomarker-driven, personalized approaches at the 2025 Carolina Neuromuscular Summit. [WATCH TIME: 2 minutes]