Neuromuscular

Brian Lin, PhD, Research Director at the Muscular Dystrophy Association, commented on the first-ever clinical trial framework for Charcot-Marie-Tooth disease, what it means for drug developers, and where the field's biomarker and endpoint evidence still needs to grow.

Nestor Galvez-Jimenez, MD, a neurologist at Baptist Health Miami Neuroscience Institute, discussed how advancements have changed the landscape of care for patients living with spinal muscular atrophy.

Regenxbio recently announced that the FDA placed RGX-121 on hold after spinal MRI abnormalities emerged in 5 participants with MPS II and does not expect a near-term BLA filing.

Dustin Gable, MD, PhD, a pediatric neuromuscular neurologist at Johns Hopkins Medicine, discussed why bulbar dysfunction remains an underrecognized burden among patients with spinal muscular atrophy.

A coalition of Charcot-Marie Tooth patient groups, clinicians, and pharma companies has published the first consensus framework for designing clinical trials in the disease.

The FDA extended its review of deramiocel for Duchenne muscular dystrophy to November 22, 2026, after accepting additional HOPE-3 data.

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

AMO Pharma reached agreement with the FDA, UK MHRA, and Health Canada on the design of a registrational study of AMO-02 for congenital myotonic dystrophy type 1, which will use hospitalization as its primary outcome measure.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending August 21, 2026.

Nicholas Streicher, MD, MPH, an assistant professor of neurology at Georgetown University, discussed the training, handoff, and coding gaps that can leave adults with spinal muscular atrophy without follow-up care.

New prespecified MINT analysis found that inebilizumab reduced exacerbations and rescue therapy use compared with placebo in adults with AChR- or MuSK-positive generalized myasthenia gravis.

Alexandra Bonner, MD, a pediatric neuromuscular neurologist at Cleveland Clinic, discussed why real-time communication between specialists is the hardest part of multidisciplinary care for patients with spinal muscular atrophy.

Oak Hill Bio announced dosing of the first participant in BEACON, a pivotal phase 3 trial evaluating rugonersen, an antisense oligonucleotide originally developed by Roche, for Angelman syndrome.

Alexandra Bonner, MD, a pediatric neuromuscular neurologist at Cleveland Clinic, discussed which specialties are essential to a spinal muscular atrophy care team and why anticipating future needs has become central to the work.

A recently published case study highlights a 17-year-old patient with Duchenne muscular dystrophy who experienced acute and delayed cardiac toxic effects under delandistrogene moxeparvovec treatment, with tissue analyses showing low-level, heterogeneous microdystrophin expression.

Stacey Tarrant, RD, LDN, discusses how earlier treatment alongside disease-modifying therapies have changed nutritional priorities in spinal muscular atrophy, including the need to optimize growth, strength, function, and body composition.

The new data from ALKIVIA marks the first phase 3 study to show statistically significant, clinically meaningful improvement in immune-mediated necrotizing myopathy, a subtype with no approved therapy.

W. Bryan Burnette, MD, chief of neurology at Nemours Children’s Health in Jacksonville, Florida, discussed why access to adult subspecialty care has become one of the largest remaining challenges in spinal muscular atrophy.

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 August 14, 2026.

CurePSP and UCSF announced enrollment of the first participant in the PSP Trial Platform, believed to be the first platform trial ever conducted specifically for progressive supranuclear palsy, designed to test multiple investigational drugs simultaneously.

Ractigen Therapeutics announced completion of enrollment and first dosing across all cohorts of its phase 2 trial of RAG-17, an investigational siRNA therapy for SOD1-mutated ALS, building on phase 1 data recently published in Nature Medicine.

Senda Ajroud-Driss, MD, director of the ALS Clinic at Northwestern Medicine, discussed why adults with spinal muscular atrophy continue to need multidisciplinary care and where the evidence still falls short.

Divya Jayaraman, MD, PhD, a pediatric neuromuscular specialist at Columbia University Irving Medical Center, discussed the comparative evidence gaps in spinal muscular atrophy and the difficulty of moving patients into adult care.

Annexon reported that all 10 patients in the first US and European cohort of its open-label FORWARD study showed rapid, clinically meaningful improvement in strength within days of a single tanruprubart infusion for Guillain-Barré syndrome.