Neuromuscular

KYV-101 shows promising efficacy in treating generalized myasthenia gravis, offering hope for durable remission through innovative CAR T-cell therapy.

Ryan Donaghy, MD, an assistant professor of neurology at Northwestern Medicine, discussed the evolving landscape of ALS clinical trials, including advances in biomarkers, genetics, and modern trial design.

Carrie Haverty, MS, CGC, NSGC president-elect, discusses the evolving role of genetic counselors, emphasizing personalized care, gene therapy advancements, and the need for multidisciplinary integration in health care.

Results of a new study funded by the ALS Association showed that the global prevalence of patients living with amyotrophic lateral sclerosis may increase by 25% by 2040.

Naji Gehchan, MD, MSc, chief medical and development officer Kyverna, discussed how KYV-101 may redefine generalized myasthenia gravis care through deep B-cell depletion and durable, drug-free remission.

Clene Nanomedicine prepares to submit an NDA for CNM-Au8, a promising ALS treatment, aiming for accelerated FDA approval by 2026.

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

Spencer Hutto, MD, associate program director of the adult neurology residency at Emory University, outlined the key diagnostic nuances, treatment considerations, and emerging therapeutic strategies shaping modern neurosarcoidosis care.

The agency has required a postmarketing observational study to monitor safety for at least 12 months following gene therapy treatment in patients with Duchenne muscular dystrophy.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending November 14, 2025.

The phase 3 FORTIFY trial of BBP-418 met its primary and key secondary interim analysis end points and demonstrated a safety profile consistent with prior studies in patients with LGMD2I/R9.

The vice president and Asset Head of Rare and Ultra-Rare Diseases at UCB provided clinical perspectives on the FDA approval of Kygevvi, the first marketed treatment specific for patients with thymidine kinase 2 deficiency (Tk2d).

A genetic counselor-only clinic enhances access to genetic testing in pediatric neurology, proving efficient and effective for timely precision diagnostics.

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.

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]