Neuromuscular

Stephanie Fradette, PharmD, senior vice president and head of Rare Neurology Department Unit at Biogen, gave commentary on the FDA designation and long-term outlook for salanersen, an investigational antisense agent for spinal muscular atrophy.

Postmortem analyses of 8 individuals with SOD1-ALS who received tofersen showed widespread drug distribution across somatic motor system tissues and reductions of up to 84% in lumbar spinal cord SOD1 protein levels.

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 July 24, 2026.

Andrew Lee, MD, PhD, cofounder and CEO of Spot Biosystems, discusses the company's extracellular vesicle platform, the first non-viral delivery of full-length dystrophin in patients with Duchenne muscular dystrophy.

Epilepsy expert Susan Duberstein, MD, explores how multidisciplinary neurocutaneous clinics improve diagnosis, surveillance, care coordination, and long-term outcomes for patients with complex neurocutaneous syndromes.

Spinogenix’s codabakalner, an investigational BK channel modulator, is currently being evaluated in CLARITY, a phase 2b/3 trial for the treatment of patients with Fragile X syndrome.

Ahead of World Brain Day, NeurologyLive explores 5 emerging strategies for preventing neurologic disease, from modifiable dementia risk factors to GLP-1 drugs, sleep science, the gut brain axis, and wearable technology.

A look at where things stand for Fragile X syndrome drug development, covering seven agents across four mechanisms as the field regroups from two recent Phase 3 setbacks.

Brett Morrison, MD, PhD, associate professor at Johns Hopkins University, recaps his PNS 2026 presentation on current and emerging biomarkers in peripheral nerve disease, from neurofilament light chain to muscle MRI and microRNA.

The FDA granted priority review to zeleciment rostudirsen for exon 51-amenable Duchenne muscular dystrophy, with a PDUFA target action date of January 21, 2027.

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 July 10, 2026.

Led by Na Tosha Gatson, MD, PhD, FAAN, director of the Center for Neuro-Oncology at the Indiana University Health Neurosciences and Cancer Institutes, this new column translates complex neuro-oncology topics into accessible, evidence-based insights.

The professor of neurology and neuroscience at Johns Hopkins University School of Medicine discussed the ongoing debate around animal models in neuropathy research, the rise of iPSC-derived systems, and why he believes both approaches are needed going forward.

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

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 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.