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New data from a Cure SMA survey showed that children treated with disease-modifying therapy soon after birth walk more often and have fewer comorbidities than those treated later.

A new Bayesian network meta-analysis found nipocalimab's serious adverse event rates compared favorably with other gMG therapies, while overall adverse event rates were comparable.

An updated open-label extension analysis and new MRI data from the phase 3 EPIDYS trial, presented at the 2026 AANEM Annual Meeting, add to the evidence for givinostat's long-term effects on disease progression.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending October 2, 2026.

Findings from the phase 3 TransportNPC study showed that Trappsol Cyclo missed the primary end point in Niemann-Pick type C, but subgroup and survival data support planned 2026 NDA.

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 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.