Neuromuscular

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.

Sandeep Rana, MD, director of the ALS Center at the Allegheny Health Network Neuroscience Institute, discussed how multidisciplinary clinics are assembled and what still limits access to coordinated care in spinal muscular atrophy.

Divya Jayaraman, MD, PhD, a pediatric neuromuscular specialist at Columbia University Irving Medical Center, discussed how multidisciplinary care teams in spinal muscular atrophy are adapting to a growing number of treatment options.

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 7, 2026.

Six FDA PDUFA decisions this fall could reshape care in Alzheimer disease, DMD, Sanfilippo syndrome, Alexander disease, and SMA, here's what clinicians need to know about the data behind each.

In recognition of SMA Awareness Month, held annually throughout August, NeurologyLive® summarized the latest literature on the multidisciplinary care approach for patients living with spinal muscular atrophy.

Elizabeth Ferluga, MD, and Kristen Fortino discuss a newly launched virtual care program, the barriers facing patients with ataxia, and the potential for remote access to specialized care to improve diagnosis, treatment, and clinical trial connectivity.

The associate professor at the University of Michigan covered the 2026 Peripheral Nerve Society Annual Meeting, highlighting emerging themes in GBS, SMA, TTR neuropathy, RFC1, and what is still keeping the field up at night.

The FDA has accepted Saol Therapeutics' resubmitted NDA for SL1009 in pyruvate dehydrogenase complex deficiency, setting a new PDUFA target action date of December 30, 2026.