Neuromuscular

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending July 3, 2026.

New phase 2b PARADIGM data showed PrimeC significantly reduced neuron-derived TDP-43 levels in patients with ALS while reinforcing previously reported improvements in disease progression and survival.

Results from the Baby-COMET study of avalglucosidase alfa in infantile-onset Pompe disease will support a planned US regulatory submission for a label extension, anticipated in the second half of 2026.

The FDA accepted supplemental applications seek to convert the accelerated approvals of casimersen and golodirsen to traditional approvals in patients with Duchenne muscular dystrophy.

The FDA will review Capricor Therapeutics' Deramiocel for the treatment of Duchenne muscular dystrophy on July 29, 2026, ahead of the therapy's PDUFA target action date of August 22, 2026.

The ongoing phase 3 RELIEVE trial investigates the efficacy and safety of remibrutinib in patients with generalized myasthenia gravis who are on stable standard-of-care treatment.

Johnson, who set the single-season NFL rushing record in 2009, went public with his diagnosis on Good Morning America, saying he hopes his story accelerates research and awareness of the disease.

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 June 26, 2026.

Following the FDA's expanded approval of efgartigimod, the neuromuscular physician at Erlanger Health Systems discusses the implications for seronegative disease, treatment sequencing, and the evolving definition of antibody-negative MG. [WATCH TIME: 5 minutes]

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 associate professor at Johns Hopkins University discussed emerging biomarkers outside of neurofilament light chain, including muscle MRI, immune-targeted markers, and microRNA research in peripheral nerve disorders. [WATCH TIME: 3 minutes]

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.

Brett Morrison, MD, PhD, associate professor at Johns Hopkins University, discussed the current landscape of biomarkers in peripheral nerve disorders and the evolving role of neurofilament light chain in clinical care and research. [WATCH TIME: 3 minutes]

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.