Neuromuscular

Clene plans to pursue accelerated FDA approval for CNM-Au8 in ALS based primarily on neurofilament light biomarker data and phase 2 studies, with a confirmatory phase 3 trial expected to follow.

Following his symposium at the 2026 AAN Annual Meeting, science educator Bill Nye discussed his connection to Friedreich ataxia, emphasizing the importance of awareness, early diagnosis, and effective communication between clinicians and patients. [WATCH TIME: 4 minutes]

Aprill Lane, US Advocacy Lead for Rare Disease at UCB, discusses a new national initiative aimed at improving nutrition access and reducing daily disease burden for patients with myasthenia gravis.

New phase 2 data indicate that dazucorilant may reduce mortality risk in amyotrophic lateral sclerosis, with sustained effects observed through 2 years, although no significant improvement in functional outcomes was reported.

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

Catch up on any of the neurology headlines you may have missed in April 2026, compiled into 1 place by the NeurologyLive® team.

The clinical professor of adult neurology at Stanford University discussed data he presented at AAN's 2026 Meeting [WATCH TIME: 4 minutes]

Amy Waldman, MD, associate professor of neurology at the Perelman School of Medicine at the University of Pennsylvania, breaks down phase 1–3 data showing zilganersen’s potential to modify disease progression in Alexander disease.

Phase 1 data presented at AAN 2026 suggest that investigational siRNA therapy RAG-17 may reduce key disease biomarkers and show early signs of clinical stabilization following a single dose in patients with SOD1-ALS.

The associate professor of neurology at the Perelman School of Medicine at the University of Pennsylvania discussed pivotal data on zilganersen, highlighting its efficacy, safety, and potential as the first disease-modifying therapy for Alexander disease.

New long-term and post hoc data presented at the 2026 AAN Annual Meeting highlighted durable efficacy, safety, and patient-centered outcomes with nipocalimab in antibody-positive generalized myasthenia gravis.

Phase 3 data presented at AAN 2026 show that IVIG improves response rates in patients with autoimmune encephalitis refractory to steroids, supporting its role as a second-line immunotherapy option.

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

The assistant professor in the Department of Chemistry and Biochemistry at University of Nevada, Las Vegas reflected on receiving the first MDA Research Momentum Award at the 2026 MDA Conference. [WATCH TIME: 2 minutes]

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending April 24, 2026.

In the phase 3 NIMBLE trial, Cemdisiran met primary and secondary endpoints, demonstrating rapid and sustained improvements in MG-ADL and QMG scores with once-every-12-week dosing in AChR-positive generalized myasthenia gravis.

In the global phase 3 PREVAIL trial, gefurulimab met primary and secondary endpoints in adults with acetylcholine receptor antibody–positive generalized myasthenia gravis, potentially offering a more convenient alternative to intravenous therapies.

Data show ofatumumab maintains a steady safety profile in relapsing MS, with low serious infections, stable labs, and durable biomarker improvements.

In a 58-week open-label extension, most patients with Parkinson disease treated with tavapadon avoided starting levodopa or required no dose increases, suggesting a potential levodopa-sparing effect.

A phase 3, double-blind study is assessing telitacicept in generalized myasthenia gravis, following prior trials that demonstrated substantial improvements in functional and strength-based clinical outcomes.

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

Semaglutide shows CNS biomarker shifts in early Alzheimer disease, lowering CSF tau and neurogranin while reshaping immune signals, as per new AAN data report.

Week-52 data from the phase 3 MINT trial show sustained improvements in MG-ADL and QMG scores with inebilizumab in patients with AChR-positive generalized myasthenia gravis.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending April 17, 2026.

In honor of International Pompe Day, held April 15, 2026, NeurologyLive reviewed recent studies on the latest research and evolving standards of care for Pompe disease.