Neuromuscular

Catch up on any of the neurology news headlines you may have missed over the course of June 2025, compiled all into one place by the NeurologyLive® team.

New findings reveal deramiocel's promising long-term benefits for boys with Duchenne muscular dystrophy cardiomyopathy, enhancing cardiac function and muscle performance.

New phase 1 data revealed salanersen's promise to slow neurodegeneration in spinal muscular atrophy, potentially paving the way for future phase 3 trials.

Edgewise Therapeutics reveals promising results for sevasemten, potentially the first approved treatment for Becker muscular dystrophy, enhancing patient outcomes.

Overviewing the major clinical trial readouts in neurology from the first half of 2025, with data that could reshape patient care.

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

Efgartigimod alfa SC is approved in Europe for patients with chronic inflammatory demyelinating polyneuropathy, following a positive recommendation from the Committee for Medicinal Products for Human Use.

Experts in ALS care and research shared their optimism about emerging treatments, aiming to deepen scientific understanding, and the potential role of advocacy in accelerating progress. [WATCH TIME: 4 minutes]

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

Experts discussed the ongoing importance of awareness, funding, and early diagnosis in amyotrophic lateral sclerosis, welcoming the return of the well-known Ice Bucket Challenge. [WATCH TIME: 5 minutes]

Test your neurology knowledge with NeurologyLive®'s weekly quiz series, featuring questions on a variety of clinical and historical neurology topics. This week's topic is on pediatric neurology.

Clinicians reflected on the unique, patient-centered format of the ALS Nexus conference and its potential role in accelerating ALS research and promoting care collaboration. [WATCH TIME: 4 minutes]

A panel of neuromuscular experts emphasized the importance of interdisciplinary care in helping improve quality of life and outcomes among patients living with ALS. [WATCH TIME: 4 minutes]

Biogen recently launched a pivotal phase 3 trial for omaveloxolone, aiming to explore its safety and efficacy in pediatric patients with Friedreich ataxia.

Experts discussed promising developments in ALS treatment, including gene-targeted therapies, emerging biomarkers, and AI-powered tools for diagnosis and patient support. [WATCH TIME: 5 minutes]

Marketed as the first drug to target both BLyS and APRIL, Telitacicept may offer a revolutionary treatment option for patients with myasthenia gravis.

Researchers uncover a latent infection model for Creutzfeldt-Jakob disease, revealing insights into its mysterious origins and potential triggers.

New long-term data support ecopipam's safety and sustained efficacy in reducing tics and improving quality of life in pediatric Tourette syndrome.

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

The director of the DMD Program at UMass Chan Medical School shared her experience at the 2025 CureDuchenne FUTURES National Conference, held May 22-25, in San Antonio, Texas. [WATCH TIME: 5 minutes]

Avidity Biosciences advances del-brax for FSHD, initiating a pivotal Phase 3 study while exploring accelerated FDA approval pathways.

Daniel Mikol, MD, PhD, chief medical officer at NervGen Pharma, provided clinical perspective on recently announced positive findings from a phase 1b/2a trial testing NVG-291 as a treatment for spinal cord injury.

The neurosurgeon at Northwestern Medicine discussed diagnostic approaches, evolving treatment strategies, and the importance of timely referral in managing peripheral nerve injuries. [WATCH TIME: 3 minutes]

New 5-year findings from the National RLS Opioid Registry presented at SLEEP 2025 highlight the long-term stability and dose trends of low-dose opioid therapy in patients with restless legs syndrome.

Members from Nuvig Therapeutics discussed how NVG-2089 could improve the CIDP treatment landscape by offering IVIG-like efficacy with a better safety and administration profile. [WATCH TIME: 3 minutes]