Neuromuscular

Updated phase 1/2 POLARIS data showed sustained reductions in seizure frequency and progressive gains in cognitive and adaptive measures following a single administration of investigational ETX101 in children with SCN1A+ Dravet syndrome.

New real-world data showed that daily corticosteroid therapy preserved motor function longer in patients with Duchenne muscular dystrophy compared with an intermittent dosing regimen.

Episode 1 features a conversation with Ahmet Hoke, MD, PhD, of Johns Hopkins Medicine, discussing the clinical debate behind keeping animal models for neuropathy research vs switching to induced pluripotent stem cell models.

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 September 4, 2026.

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Aaron Zelikovich, MD. [LISTEN TIME: 18 minutes]

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

FDA approved Zanvastro (zilganersen), the first therapy to directly target the GFAP protein buildup that drives Alexander disease, based on a phase 3 trial showing improved walking speed and motor function versus untreated controls.

The phase 3 Aspire study of apazunersen did not meet its primary cognition end point or key secondary end point in Angelman syndrome.

The neurologist at the UCSF Edward and Pearl Fein Memory and Aging Center discusses how the first platform trial in progressive supranuclear palsy could accelerate the evaluation of potential disease-modifying therapies. [WATCH TIME: 4 minutes]

In recognition of SMA Awareness Month, NeurologyLive® spoke with neuromuscular experts about how multidisciplinary care is built, where coordination breaks down, and what changes as patients live longer.

In recognition of SMA Awareness Month, NeurologyLive® spoke with neuromuscular experts about how multidisciplinary care is built, where coordination breaks down, and what changes as patients live longer.

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

Catch up on any of the neurology headlines you may have missed from last week, compiled into 1 place by the NeurologyLive® team.

In honor of SMA Awareness Month, held annually throughout August, get caught up on the latest news in spinal muscular atrophy from 2026, 5 headlines compiled in one place by the NeurologyLive® team.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending August 28, 2026.

In recognition of SMA Awareness Month, held annually throughout August, NeurologyLive® reviews the investigational agents currently moving through clinical development for spinal muscular atrophy.

The phase 3 clinical program for salanersen, an investigational intrathecally administered antisense oligonucleotide, will test the efficacy of the agent across infants, adolescents and adults living with SMA.

Cellenkos’ investigational allogeneic, cord blood-derived T-regulatory cell therapy is being evaluated in a phase 1/1b study in ALS, with early findings showing reductions in plasma neurofilament light chain and increases in IL-10.

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.