Neuromuscular

Two-year data from the phase 2 DAZALS trial showed a lower risk of death with dazucorilant 300 mg/day vs placebo in ALS, although the study did not meet its functional primary end point.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending November 6, 2026.

Servier reported that sevasemten did not meet its primary or secondary endpoints in the pivotal GRAND CANYON trial for Becker muscular dystrophy.

QurAlis reported interim ANQUR trial results showing target engagement, a biomarker signal, and a trend toward slower functional decline with QRL-201 in ALS.

The 500-participant study will assess whether pridopidine, an investigational sigma-1 receptor agonist, can slow functional decline and improve survival in patients with early, rapidly progressing amyotrophic lateral sclerosis.

Kiril Kiprovski, MD, clinical professor of neurology and director of the Division of Neuromuscular Medicine at NYU Langone, reviews the clinical clues, bedside tests, and referral considerations that help eye care clinicians identify Lambert-Eaton myasthenic syndrome in patients with fluctuating ptosis or diplopia.

uniQure presented final safety and biomarker results from the discontinued EPISOD1 trial of AMT-162, an investigational gene therapy for SOD1-associated ALS.

Richard Nowak, MD, director of the Yale Myasthenia Gravis Clinic and global principal investigator of the MINT trial, discusses open-label extension findings for inebilizumab, a CD19-directed B cell depleting therapy, in generalized myasthenia gravis.

AL-S Pharma outlined the design of AP101-03, a planned phase 3 trial of AP-101 in sporadic and SOD1-associated ALS, at the 2026 NEALS Annual Meeting.

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

Interim phase 1b/2a data showed all patients treated with high- or low-dose BB-301 for oculopharyngeal muscular dystrophy met formal response criteria.

Although ocular signs in LEMS are typically mild and appear late, recognizing them alongside proximal weakness and autonomic symptoms can speed diagnosis and earlier detection of an associated lung cancer.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending October 30, 2026.

Shaida Khan, DO, associate professor of neurology at UT Southwestern Medical Center, reflected on a recent NeurologyLive Clinical Forum on generalized myasthenia gravis and discussed how FcRn and complement inhibitors are reshaping treatment algorithms for the disease.

Paul Melmeyer, MPP, Executive Vice President of Public Policy and Advocacy at the Muscular Dystrophy Association, discusses the Neuromuscular Advocacy Collaborative, its growing year-round presence, and what the group hopes to accomplish heading into the 2027 MDA Clinical and Scientific Conference.

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 October 23, 2026.

The FDA set a first quarter 2027 goal date for troriluzole in spinocerebellar ataxia and plans an advisory committee after a 2025 complete response letter.

Clene has filed an NDA seeking accelerated approval of CNM-Au8 for ALS following 2 missed primary trial end points and years of evolving FDA guidance.

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

Leonard Dragone, MD, PhD, Disease Area Leader at Johnson & Johnson, discusses how nipocalimab is being incorporated into generalized MG care, what long-term data show, and where J&J sees the therapy’s role evolving.

Pivotal AFFINITY DUCHENNE results showed microdystrophin expression above the prespecified threshold in 93% of evaluable participants, with preliminary functional findings favoring treatment over external controls.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending October 16, 2026.

Three-year extension data from the phase 3 IB1001-301 trial show levacetylleucine slowed disease progression in both adults and children with Niemann-Pick disease type C.

Adults with generalized myasthenia gravis who are anti-acetylcholine receptor antibody positive now have a second self-administration option for the complement inhibitor zilucoplan.