Neuromuscular

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

Test your neurology knowledge with NeurologyLive®'s weekly quiz series, featuring questions on a variety of clinical and historical neurology topics. This week's topic is on RNA therapeutics for neuromuscular disorders.

AL-S Pharma's AP-101 shows promising safety and efficacy in ALS patients, paving the way for future treatments targeting misfolded SOD1 proteins.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending September 5, 2025.

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

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending August 29, 2025.

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.

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.

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 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.

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.

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.