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The designation for the investigational brain-penetrant IDH1 inhibitor comes as the pivotal phase 3 SIGMA study evaluates its use in patients with high-risk IDH1-mutant astrocytoma.

Dustin Gable, MD, PhD, a pediatric neuromuscular neurologist at Johns Hopkins Medicine, discussed why bulbar dysfunction remains an underrecognized burden among patients with spinal muscular atrophy.

A coalition of Charcot-Marie Tooth patient groups, clinicians, and pharma companies has published the first consensus framework for designing clinical trials in the disease.

The FDA extended its review of deramiocel for Duchenne muscular dystrophy to November 22, 2026, after accepting additional HOPE-3 data.

The FDA has cleared Roche's Elecsys pTau217, a blood test that uses a single biomarker to both rule in and rule out amyloid pathology in adults being evaluated for Alzheimer disease, across both primary and specialty care settings.

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 Huntington disease!

AMO Pharma reached agreement with the FDA, UK MHRA, and Health Canada on the design of a registrational study of AMO-02 for congenital myotonic dystrophy type 1, which will use hospitalization as its primary outcome measure.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending August 21, 2026.

Nicholas Streicher, MD, MPH, an assistant professor of neurology at Georgetown University, discussed the training, handoff, and coding gaps that can leave adults with spinal muscular atrophy without follow-up care.

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

New prespecified MINT analysis found that inebilizumab reduced exacerbations and rescue therapy use compared with placebo in adults with AChR- or MuSK-positive generalized myasthenia gravis.

Full results from the phase 3 TEMPO-2 trial, published in Lancet Neurology, showed that flexible-dose tavapadon significantly improved motor symptoms and daily function in people with early Parkinson disease.