Neuromuscular

At the 2023 AAN Annual Meeting, the neuromuscular disease specialist and professor of neurology at the University of Kansas Medical Center talked about the phase 3 study investigating ataluren for Duchenne muscular dystrophy. [WATCH TIME: 5 minutes]

Leo H. Wang, MD, PhD, FAAN, associate professor of neurology, University of Washington Medical Center, talked about the phase 2 study of losmapimod for facioscapulohumeral muscular dystrophy presented at the 2023 AAN Annual Meeting.

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 headache and migraine.

The FDA is set to make a final decision later this month and, if approved, SRP-9001 would be the first gene therapy specific to treat Duchenne muscular dystrophy.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending May 12, 2023.

To date, the enzyme replacement therapy had been studied in a comprehensive clinical development program that comprised more than 140 patients with Fabry disease with up to 7.5 years of follow up treatment.

Nicholas Johnson, MD, MSci, FAAN, division chief of neuromuscular disorders and vice chair of research, Virginia Commonwealth University, provided insight on promising phase 1/2 data of AOC 1001 in myotonic dystrophy type 1.

Melanie Leitner, PhD, consultant and founder of Accelerating NeuroVentures, talked about the findings from regimen D of the HEALY ALS platform trial assessing pridopidine.

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 epilepsy and seizure disorders.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending May 5, 2023.

The associate chief science officer at Kennedy Krieger Institute shared thoughts on the state of pediatric neurology and the significance of being named winner of the Sydney Carter Award. [WATCH TIME: 3 minutes]

Designed for self-directed use in a patient’s home or a healthcare facility, the Cumulus EEG device has potential to change the clinical trial landscape, allowing researchers to conduct more remote studies.

After 48 months of follow up, cipaglucosidase alfa/miglustat in cohorts of ambulatory patients with Pompe disease had an overall safety profile similar to the approved enzyme replacement therapy.

The 2-part study is intended to first establish safety and tolerability of multiple doses of CK0803, followed by efficacy analyses on several known ALS outcome measures.

A group of experts in the care of patients with neurological conditions—Ali Jannati, MD, PhD; Madhav R. Thambisetty, MD, PhD; Daniel Ontaneda, MD, PhD; Peter J. Goadsby, MD, PhD, DSc; Andrew Russman, DO—shared their perspectives on hot topics of treatment and management in neurology from the 2023 American Academy of Neurology Annual Meeting.

The director of Cleveland Clinic’s Epilepsy Center at the Cleveland Clinic Neurological Institute provided perspective on the in-depth testing neurologically healthy patients in the Brain Study will undergo. [WATCH TIME: 3 minutes]

Total disease burden, measured by the Facioscapulohumeral Muscular Dystrophy Health Index, was reduced by 19% over a 24-week period on recombinant human growth hormone.

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

Experts review the state of SMA diagnosis and medications, highlighting factors that guide treatment selection in North America and South America.

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 the history of the American Academy of Neurology.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending April 28, 2023.

The president elect of the Association of University Professors of Neurology discussed the benefits of working in neurology and the ways to not only attract new clinicians, but keep those already in the field. [WATCH TIME: 3 minutes]

The director of the Massachusetts General Hospital ALS Care Center discussed the importance of effective disease-modifying therapies for ALS and the potential of CNM-Au8 in the management of the disease. [WATCH TIME: 2 minutes]

Minoryx's leriglitazone showed a decrease in matrix metalloproteinase-9 concentrations in pediatric patients with cerebral adrenoleukodystrophy, according to a 24-week analysis.

After 66 weeks of treatment, patients on eplontersen showed sustained reduction in TTR concentration, halted progression of neuropathy impairment, and improved quality of life.

A. Gordon Smith, MD, FAAN, talks about the importance of collaboration and education amid advances in myasthenia gravis treatment, from new formulations and routes of administration to innovative targets.

A. Gordon Smith, MD, FAAN, describes the landscape of investigational therapies for myasthenia gravis, including plasma cells, CAR-T, antigen-specific tolerance.

The president elect of the Association of University Professors of Neurology provided thoughts on the promising concepts in neurology and the reasons young medical professionals should join the field.