Neuromuscular

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.

All 10 REVEAL Part A participants gained or regained at least 1 developmental milestone after a single intrathecal TSHA-102 dose, with no treatment-related SAEs reported.

The phase 1 ASCEND trial is testing a single intrathecal dose of the investigational AAV9 gene therapy INS1201 in young boys with Duchenne muscular dystrophy.

The designation follows phase 2 ClearMEMory data in which the oral NMDA receptor antagonist improved objective cognitive performance over placebo at 12 weeks.

A post hoc analysis of the phase 2/3 NPC002 trial presented at CNS 2026 showed that the benefit of arimoclomol plus miglustat in Niemann-Pick disease type C emerged as early as 3 months after treatment initiation.

The professor of neurology and program director of Neuromuscular Medicine at UC Irvine, discusses phase 3 NIMBLE trial findings for cemdisiran in generalized myasthenia gravis, including its novel siRNA mechanism, efficacy, and hospitalization data.

New 48-week phase 3 data show many patients with generalized myasthenia gravis reached minimal symptom expression on telitacicept, and most who did sustained it.

New analyses found differences in functional outcomes over 2 years among patients aged 8 to 12 years treated with delandistrogene moxeparvovec, while separate findings characterized micro-dystrophin expression in children younger than 3 years.

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 9, 2026.

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

Christen Kutz, PhD, PA-C, discusses a retrospective survey of neurology providers examining why patients with generalized myasthenia gravis are transitioning to nipocalimab and what real-world outcomes data suggest about its role in the treatment landscape.

At a NeurologyLive Clinical Forum in Philadelphia, Jessica D. Schulte, MD, PhD, of NYU Grossman School of Medicine, discussed how earlier recognition and systemic therapy options are changing the treatment paradigm for patients with NF1-associated plexiform neurofibromas.

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

PROPEL will test imeroprubart, an investigational FcRn blocker, against placebo in adults with mild to severe generalized myasthenia gravis.

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.