Neuromuscular

A study reveals the intricate decision-making process caregivers face when considering gene therapy for Duchenne muscular dystrophy, emphasizing shared input and personal experiences.

Although their knowledge, experience, and access to genetic counselors varied, most licensed OB/GYNs across Ohio reported routinely offering carrier screening for spinal muscular atrophy.

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

Neurology News Network for the week ending November 8, 2025. [WATCH TIME: 4 minutes]

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending November 7, 2025.

A new study presented at NSGC 2025 showed that patients living with amyotrophic lateral sclerosis considering medical aid in dying often experienced emotional and logistical challenges with the process.

A recent study highlights the link between genetic neurodevelopmental disorders and increased gastrointestinal issues in pediatric patients, emphasizing the need for targeted care.

Spingogenix presents promising data on SPG302, a daily tablet showing cognitive improvements in ALS patients with excellent safety profiles.

The chief medical officer at Dyne Therapeutics discussed the scientific rationale and clinical progress of z-basivarsen, an antisense oligonucleotide therapy designed to correct mis-splicing in myotonic dystrophy type 1. [WATCH TIME: 5 minutes]

Feedback from the FDA allows CervoMed to move forward with the proposed primary and secondary end point of the phase 3 trial assessing neflamapimod in dementia with Lewy bodies.

FDA issues a complete response letter for Biohaven's troriluzole, a potential treatment for spinocerebellar ataxia, citing data concerns.

The associate professor of physical medicine and rehabilitation at Virginia Commonwealth University discussed his passion for educating others about electrodiagnosis and ultrasound in neuromuscular disorders.

The neuroscience program coordinator at Loyola University Medical Center talked about a recent global nurse survey for those caring for patients with generalized myasthenia gravis. [WATCH TIME: 5 minutes]

A phase 1/2 trial explores YTB323, a CAR T cell therapy, for treating generalized myasthenia gravis, aiming to enhance patient outcomes and safety.

UCB's Kygevvi becomes the first FDA-approved treatment for thymidine kinase 2 deficiency, offering hope to patients with this rare neuromuscular disorder.

The associate professor of clinical neurology at Keck Medicine of USC discussed advancements in therapeutics, the growing focus on biomarkers, and ongoing challenges in myasthenia gravis. [WATCH TIME: 4 minutes]

The associate professor of neurology at Yale School of Medicine discussed 52-week data from the phase 3 MINT trial of inebilizumab in patients with generalized myasthenia gravis. [WATCH TIME: 5 minutes]

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

The head of Rare Diseases US at UCB talked about the company’s latest data on rozanolixizumab and zilucoplan in patients living with generalized myasthenia gravis. [WATCH TIME: 3 minutes]

The chief medical officer at Mandos Health discussed new CNS 2025 data on adrabetadex in infantile-onset Niemann-Pick type C and the growing evidence of its potential disease-modifying impact.

Neurology News Network for the week ending November 1, 2025. [WATCH TIME: 3 minutes]

A groundbreaking trial compares nipocalimab and efgartigimod for treating generalized myasthenia gravis, promising new insights for patient care.

A perspective model aimed noted that introducing efgartigimod alfa for patients with chronic inflammatory demyelinating polyneuropathy incurs more expected costs.

Patients with generalized myasthenia gravis reported a positive experience with rozanolixizumab self-administration, with a preference for the manual push method.

An open-label extension trial testing the efficacy of nipocalimab in patients transitioning from placebo revealed continued improvement in MG-Activities of Daily Living scores over a long-term period.