Neuromuscular

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.

In honor of Fragile X Syndrome Awareness Day, held annually on July 22, the professor of psychiatry at Cincinnati Children's Hospital outlined where testing for the disorder still falls short. [WATCH TIME: 6 minutes]

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.

The director of the Division of Neuroscience at IRCCS Istituto Ortopedico Rizzoli discusses why targeting shared biologic pathways, rather than individual mutations, may represent a promising therapeutic strategy for Charcot-Marie-Tooth disease. [WATCH TIME: 2 minutes]

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.

Matthew Evans, BM, BCh, DPhil, a consultant neurologist at the University of Oxford, discusses the diagnostic approach to small fiber neuropathy and the challenges of confirming immune-mediated disease. [WATCH TIME: 3 minutes]

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.

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.