Neuromuscular

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.

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

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

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 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.

A recent study reveals the significant economic burden of Angelman syndrome, highlighting rising healthcare costs and ongoing therapeutic developments.

A post hoc analysis of the phase 3 EPIDYS trial presented at CNS 2025 revealed that givinostat delayed disease progression in 2 distinct age groups of patients with Duchenne muscular dystrophy.

A recent analysis highlights the link between vastus lateralis fat fraction and treatment outcomes in Duchenne muscular dystrophy patients using givinostat.

Yuichi Iwaki, MD, PhD, chief executive officer at MediciNova, spoke on ibudilast’s mechanism of action, its role in the COMBAT-ALS trial, and its potential impact across neurodegenerative disease care.

In the MOXIe open-label extension study presented at MDS 2025, omaveloxolone treatment was associated with no significant disease progression in bulbar function or upper limb coordination.

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

Nipocalimab receives positive EMA recommendation, potentially transforming treatment for generalized myasthenia gravis with significant clinical trial results.

The president of the LGMD Foundation discussed raising awareness, strengthening advocacy, and the lived experience of navigating limb-girdle muscular dystrophy.

Melissa Spencer, PhD, discussed the rare and complex landscape of limb girdle muscular dystrophy, highlighting research challenges, safety considerations in gene therapy, and the need for natural history studies and biomarkers.