Neuromuscular

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

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.

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.