Neuromuscular

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.

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]

The professor of neurology at the University of Texas Health Science Center San Antonio provided clinical insights on the genetic basis of oculopharyngeal muscular dystrophy, as well as the primary challenges in diagnosing and treating the condition. [WATCH TIME: 5 minutes]

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

Neurology News Network. for the week ending March 29, 2025. [WATCH TIME: 3 minutes]

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

The former FDA commissioner spoke on the need for policy and funding efforts for neuromuscular disorders, the stress on the clinical care system, and the need to acknowledge healthcare inequities. [WATCH TIME: 4 minutes]

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.

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

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.

Neurology News Network. for the week ending March 22, 2025. [WATCH TIME: 3 minutes]

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending March 21, 2025.

The professor of pediatrics and division chief of Medical Genetics at Duke University gave clinical insight on a presentation on the advancements and limitations of enzyme replacement therapy for Pompe Disease. [WATCH TIME: 3 minutes]

Marketed as Amvuttra, the Alnylam Pharmaceuticals RNA-interference treatment is now approved for an additional patient population of 150,000 in the US, and more than 300,000 worldwide.