Neuromuscular

Beth Stein, MD, director of neuromuscular diseases at St. Joseph’s Health, shared insights on the impact of a newly approved pre-filled syringe formulation of efgartigimod, highlighting its potential to transform care delivery.

In a late-stage trial, telitacicept demonstrated significant and rapid clinical improvements in gMG, achieving notable symptom reductions, improved quality of life, and strong safety outcomes.

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Patients treated with efgartigimod for myasthenia gravis were found to have a higher incidence and severity of infections compared to those treated with immunoglobulins.

The phase 1 LUMINA trial evaluates Amylyx Pharmaceuticals' AMX0114, an antisense oligonucleotide targeting calpain-2, as a potential therapy for ALS by focusing on safety, tolerability, and biomarkers tied to disease progression.

Phase 2b trial results showed Descartes-08, a CAR-T therapy, achieved sustained efficacy and a strong safety profile in myasthenia gravis, with notable responses in biologic-naïve patients and no major safety concerns over 12 months.

Cleveland Clinic’s $1.1 billion Neurological Institute, opening in 2027, will revolutionize neurological care with a state-of-the-art, patient-centered design integrating advanced technology and innovative care solutions.

The prefilled syringe is approved as a 20-to-30-second subcutaneous injection, where patients are able to self-inject following proper instruction in subcutaneous injection technique.

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

Cassandra Gorsuch, PhD, chief scientific officer at Precision Biosciences, discussed the company’s ARCUS gene editing platform, its innovative approach to treating Duchenne muscular dystrophy, and promising preclinical data presented at MDA 2025.

Newly presented open-label extension results at AAN 2025 suggest that investigational ZYN002, a cannabidiol gel, may help improve irritability in children and adolescents living with Fragile X syndrome.

Denali Therapeutics has begun submitting a BLA for tividenofusp alfa as a potential Hunter syndrome treatment, supported by promising biomarker reductions in clinical trials.

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Presented at the 2025 AAN Annual Meeting, findings from a phase 3 study showed that a single 30 mg/kg dose of ANX005 significantly improved health outcomes in Guillain-Barré syndrome, with benefits seen as early as week 1 and sustained over 6 months.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending April 4, 2025.

The European Medicines Agency has requested a pause on 3 clinical trials of FDA-approved gene therapy Elevidys following the death of a patient who suffered acute liver failure after treatment.

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Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending March 28, 2025.

WVE-N531, an investigational exon-skipping therapy, showed promising 48-week results in the FORWARD-53 study, marking the first-ever improvement in muscle health for patients with Duchenne muscular dystrophy, with plans for an NDA filing in 2026.

The HARBOR study is an ongoing phase 3 global study focused on assessing del‑desiran, formerly known as AOC 1001, which aims to treat the underlying cause of DM1.

The senior vice president of research at The ALS Association discussed insights into neural regeneration therapies and their potential to restore function in neurodegenerative diseases.

The associate professor of pediatric neurology and genetics at the University of Alabama Birmingham provided clinical insights on the key signaling pathways currently being targeted in the treatment of muscular dystrophies. [WATCH TIME: 4 minutes]

Neurizon Therapeutics announced updates to the HEALEY-ALS trial and promising NUZ-001 results, signaling a major step forward in ALS treatment research.

Pooled safety data of delandistrogene moxeparvovec, a gene transfer therapy for Duchenne muscular dystrophy, suggests a manageable tolerability profile up to 5 years of follow-up.

Findings from a phase 2 study of nomlabofusp, a novel investigational therapy for Friedreich ataxia, demonstrated an increase in tissue frataxin levels and metabolic improvements.

Results from a recent phase 3 study presented at the 2025 MDA Conference reported that oral edaravone remained well tolerated in patients with ALS over 96 weeks, with no new safety concerns.

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Despite facing mental health concerns with anger, aggression, or irritability, among others, slightly less than one-fourth of patients with DMD utilized psychosocial services such as counseling or therapy.

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 the MDA Clinical & Scientific conference.

The phase 2 CANYON trial findings presented at the 2025 MDA conference highlight sevasemten’s potential in reducing muscle injury biomarkers in Becker muscular dystrophy.