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Early Spinal Muscular Atrophy Treatment Tied to Milder Pediatric Phenotype

Nipocalimab’s Safety Profile Slightly Favored Over Other Myasthenia Gravis Therapies, Indirect Comparison Shows

Givinostat Data Point to Delayed Loss of Ambulation, Preserved Muscle Tissue in DMD

NeurologyLive® Friday 5 — October 2, 2026

Trappsol Cyclo Misses Primary End Point in Phase 3 Trial for Niemann-Pick Disease Type C

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The professor of neurology at the University of Lyon and 2026 Rachel Horne Prize Winner discussed pregnancy counseling, therapeutic inertia, and overlooked women's health issues in multiple sclerosis.

The Accelerating Access to Critical Therapies for ALS Reauthorization Act of 2026 extends federal support for ALS research and expanded access to investigational therapies, with additional provisions addressing clinical trial oversight and regulatory planning.

New 12-month interim data showed functional gains with BBP-418 vs placebo, consistent benefit across genotype, age, and baseline pulmonary function subgroups, and a safety profile comparable to placebo.

Real-world 12‑month results from WeSMA, a phase 4 study, showed that oral risdiplam is well tolerated in adults with SMA, with stable motor function and consistent safety findings.

A phase 4 study is evaluating whether adults with chronic inflammatory demyelinating polyneuropathy can transition from intravenous immunoglobulin to subcutaneous efgartigimod PH20 without first experiencing disease worsening.

A new phase 4 trial, OCTAGON, will test a rapid, predefined corticosteroid-tapering schedule in patients with generalized myasthenia gravis treated with ravulizumab.

PETUNIA, a new global pharmacovigilance study, will track pregnancy, maternal, and infant outcomes among patients with gMG exposed to nipocalimab.

In a pivotal phase 3 study, once-daily tasimelteon 20 mg shifted sleep onset 37.1 minutes earlier than placebo in adults with delayed sleep-wake phase disorder.

Findings from the phase 1/2 RESET-MG trial, presented at AANEM 2026, showed that 10 of 13 patients with refractory generalized myasthenia gravis had clinically meaningful MG-ADL improvements after a single infusion of rese-cel.

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Katherine Matthews, MD. [LISTEN TIME: 17 minutes]

The FDA accepted fenebrutinib's new drug application under priority review for relapsing and primary progressive MS, based on 3 phase 3 trials comparing the BTK inhibitor with teriflunomide and ocrelizumab.

A new study will follow adults with chronic inflammatory demyelinating polyradiculoneuropathy who start subcutaneous efgartigimod in routine practice in the US and Germany.

The chairman of the Neurology Service Line at Baptist Health in Kentucky discussed PREVIEW trial findings for gefelorilimab in generalized myasthenia gravis, including efficacy, safety, and what the subcutaneous C5 inhibitor could offer patients if approved.

Phase 3 trials EMNERGIZE and EMVIGORATE will evaluate the efficacy and safety of intravenous empasiprubart in adults with chronic inflammatory demyelinating polyradiculoneuropathy.

EpilepsyGTx and Viralgen have entered a manufacturing collaboration to support development of EPY201, an investigational AAV9-based gene therapy for focal refractory epilepsy planned to enter first-in-human testing in 2027.

























