Neuromuscular

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.

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.

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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.

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.

Data showed that efgartigimod led to greater improvements in disease activity compared with placebo in idiopathic inflammatory myopathy, and improvements in seronegative myasthenia gravis.

An analysis of the first 110 patients screened for the ARISE study showed that an independent adjudication committee confirmed the diagnosis of CIDP in nearly 3-quarters of cases.

Experts shared their clinical perspectives on trending topics in the treatment and management of multiple sclerosis at the 54th Child Neurology Society (CNS) Annual Meeting, held October 8-11, 2025.

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New findings reveal early treatment with Trappsol Cyclo shows promise for young patients with Niemann-Pick disease type C1, enhancing safety and efficacy.

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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 deep brain stimulation for Parkinson disease.

At the 2025 CNS Annual Meeting, Craig Press, MD, discussed tailoring neuroICU treatment for pediatric patients, advances in seizure management, and how multidisciplinary collaboration can improve outcomes.

Apitegromab, an invesigational muscle-targeting treatment, showed promising results in improving motor function among young patients with SMA in a phase 2 trial.

Denali Therapeutics prepares for potential FDA approval of tividenofusp alfa, a groundbreaking therapy for Hunter syndrome, with a revised review date set.

Trofinetide shows significant real-world improvements in Rett syndrome symptoms for pediatric and adult patients, enhancing communication and quality of life.

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

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 gene therapy for Duchenne muscular dystrophy.

A case series reveals safe use of vamorolone and prednisolone during microdystrophin gene therapy for Duchenne muscular dystrophy in two boys.

Catch up on any of the neurology news headlines you may have missed in September 2025, compiled into 1 place by the NeurologyLive® team.