Neuromuscular

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending June 26, 2026.

REGENXBIO expects to resubmit the BLA for clemidsogene lanparvovec in Q3 2026 after the FDA confirmed existing CAMPSIITE data are sufficient for accelerated approval consideration.

The professor of neuroscience at the University of Cambridge discussed the biology of programmed axon death, the emergence of SARM1-targeted therapies, and the challenges of translating this pathway into treatments for neurologic conditions.

The FDA has accepted Regeneron Pharmaceuticals’ new drug application of cemdisiran's for generalized myasthenia gravis under priority review, with a target action date of November 2026.

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

Matthew Evans, BM, BCh, DPhil, consultant neurologist at Imperial College Healthcare NHS Trust, discusses emerging evidence for immune-mediated small fiber neuropathy, current diagnostic challenges, and the need for better clinical trials and biomarkers.

Phase 3 data showed a 53% reduction in relapse risk versus placebo, and ecopipam could become the first non-antipsychotic option ever approved for Tourette syndrome.

The phase 3 EMNERGIZE study evaluates whether the complement C2 inhibitor empasiprubart can improve disability and functional outcomes in adults with chronic inflammatory demyelinating polyneuropathy.

A preclinical study found that SP16, an LRP1 agonist, reduced mechanical hypersensitivity and cold hyperalgesia in paclitaxel-treated mice in an LRP1-dependent manner, implicating Schwann cell LRP1 as a potential therapeutic target in CIPPN.

A randomized, placebo-controlled study will assess whether weekly subcutaneous imeroprubart can reduce relapse risk in patients with chronic inflammatory demyelinating polyneuropathy despite standard-of-care treatment.

New phase 2 data showed that riliprubart was associated with sustained improvements in patient-reported outcomes at 76 weeks in patients with chronic inflammatory demyelinating polyradiculoneuropathy.

The MAGNAZ trial, the first study evaluating zanubrutinib added to rituximab in anti-MAG polyneuropathy, reported baseline characteristics of 32 enrolled patients at PNS 2026, with efficacy results to follow.

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

A real-world comparative study found efgartigimod produced similar GBS disability score improvement to plasma exchange at 4 weeks, with significantly greater MRC sum score recovery at weeks 8 and 12, and a comparable safety profile.

Analysis of 1420 patients from the International GBS Outcome Study found no significant difference in GBS disability scores between IVIg and plasma exchange at 4 or 26 weeks after adjusting for key clinical covariates.

An exploratory analysis of the phase 3 ADHERE trial found serum NfL levels correlated with CIDP disease activity and declined with efgartigimod treatment in patients with elevated baseline levels.

The CAPTIVATE trial evaluates claseprubart, an active C1s inhibitor, across a broad CIDP population including SOC responders, refractory patients, and treatment-naive adults, with time to relapse as the primary endpoint in the double-blind phase.

Despite missing its primary end point, the SYNAPSE-CMT trial showed improvements in muscle strength and motor function with ignaseclant treatment in patients with Charcot-Marie-Tooth disease.

A phase 4 trial underway in the U.S. is examining whether patients with CIDP on stable IVIg can transition to efgartigimod PH20 SC within one week of their last infusion, without requiring documented disease worsening first.

Sanofi stops the phase 3 MOBILIZE study of riliprubart in chronic inflammatory demyelinating polyneuropathy because of insufficient likelihood of efficacy, with no safety signals reported.

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

Jinsy A. Andrews, MD, Director of the ALS Clinic and Director of Clinical Trials at NYU Langone Health, discussed the evolving ALS treatment landscape, including emerging gene-targeted therapies, and the next generation of disease biomarkers.

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

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 May 29, 2026.