Neuromuscular

The former FDA commissioner discussed the collaborative power of the neuromuscular community and the challenges of implementing emerging therapies in a rapidly evolving care landscape. [WATCH TIME: 4 minutes]

The chief scientific officer at Avidity Biosciences details the mechanism, early results, and future plans for Del-zota, an investigational therapy targeting exon 44 in Duchenne muscular dystrophy.

Using discovery and validation cohorts, plasma miRNA-214 levels were shown to correlate with disease severity and were also associated with plasma neurofilament light and CSF inflammatory cytokines.

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.

The chief scientific officer at Satellos Bioscience discussed phase 1 findings on SAT-3247, an oral AAK1 inhibitor, highlighting its safety, pharmacokinetics, and early translatability in Duchenne muscular dystrophy. [WATCH TIME: 7 minutes]

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.

The senior director of mission programs at The ALS Association discussed how the thinkALS Toolkit can aid general neurologists in identifying and referring suspected cases more efficiently. [WATCH TIME: 5 minutes]

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

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.

Neurology News Network. for the week ending April 12, 2025. [WATCH TIME: 3 minutes]

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 for a 20- to 30-second subcutaneous injection, and patients can self-inject following proper instruction in the 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.

The professor of neurology at the University of Texas Health Science Center San Antonio discussed some of the challenges with therapeutic development for OPMD, as well as advances and barriers to gene therapy approaches. [WATCH TIME: 4 minutes]

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.

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

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.

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Daniel Harrison, MD. [LISTEN TIME: 20 minutes]

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.

The senior vice president of research at The ALS Association talked about a multidisciplinary session presented at the 2025 MDA Conference that highlighted diverse strategies for neural repair, from pharmacological targets to noninvasive brain stimulation. [WATCH TIME: 6 minutes]