Neuromuscular

Avidity Biosciences anticipates to provide new cohort data from the phase 1/2 EXPLORE44 trial assessing AOC 1044 in Duchenne muscular dystrophy mutations amenable to exon 44 skipping in the second half of 2024.

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 Huntington disease.

BIIB105, an antisense oligonucleotide, failed to demonstrate impact on outcomes of function, breathing, and strength, in addition to no impact on neurofilament light.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending May 17, 2024.

Solid Biosciences is expecting to provide initial safety data on SGT-003 from the first couple of pediatric patients with Duchenne muscular dystrophy enrolled in the phase 1/2 INSPIRE Duchenne trial in mid-2024.

Treatment with rozanolixizumab was safe among patients with CIDP, resulted in reduced immunoglobulin levels as expected, but did not have an impact on efficacy.

Cure SMA introduces tools aiding in SMA clinical trial navigation and participation empowerment.

Lindsey Lee Lair, MD, a neurologist and vice president of clinical development at Biohaven Pharmaceuticals, provided insight on an ongoing pivotal trial assessing the impacts of taldefgrobep alfa, a myostatin inhibitor, in patients with SMA.

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 cluster headache.

A recent study recognized anti-DAGLA autoantibodies in the cerebrospinal fluid of a small group of patients, suggesting that it could be a potential biomarker for diagnosing cerebellar ataxia.

Neurology News Network. for the week ending May 11, 2024. [WATCH TIME: 4 minutes]

Avidity Biosciences is anticipated to initiate its phase 3 HARBOR trial assessing AOC 1001 as a treatment for patients with myotonic dystrophy type 1 in the second quarter of 2024.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending May 10, 2024.

Catch up on any of the neurology news headlines you may have missed over the course of April 2024, compiled all into one place by the NeurologyLive® team.

Findings from a recent study suggest that the presence of negative myoclonus may serve as a diagnostic clue for progressive myoclonus ataxia, suggesting the presence of genetic disorders.

Subgroup analyses support PrimeC’s potential in ALS treatment, aiming to inform upcoming trials for efficacy.

As the company gains more information about the patient death, dosing in the other phase 3 trial, CIFFREO, has been paused until further notice.

The associate director, Patient Reported Outcomes, Johnson & Johnson, provided clarity on a poster presentation from AAN 2024 examining the most critical aspects to CIDP disease severity.

Expert neurologists share thoughts on how to improve patient/physician communication and how listening to the patient needs will aid in managing MG.

Drs Howard, Silvestri, Vu, Habib, and Stein comment on the role of pharmacists in managing MG, emphasizing educating both the pharmacists and the patients.

Expert neurologists discuss patient-focused care when it comes to treating MG, also highlighting clinical trials.

Despite failing to meet the primary end point, AMO-02 achieved clinically and statistically significant benefit in various functional and objective assessments in the phase 2/3 REACH-CDM trial.

The vice president, Autoantibody Portfolio and Maternal Fetal Immunology Disease Area Leader, Johnson & Johnson, provided perspective on the unique molecular structure of nipocalimab seen across nonclinical and clinical studies. [WATCH TIME: 3 minutes]

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 treatment-refractory epilepsy.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending May 3, 2024.

The associate chair for technology and innovation at the NYU Grossman School of Medicine provided commentary on a study from AAN 2024 that examined the driving factors behind burnout in neurology departments. [WATCH TIME: 4 minutes]

Recent preclinical findings revealed SBT-589's potential to treat cardiomyopathy in Friedreich ataxia by targeting mitochondrial dysfunction.

Data from the phase 2 open label extension study investigating subcutaneous injections of nomlabofusp for Friedreich ataxia is expected in the fourth quarter of 2024.