Neuromuscular

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.

In single-ascending dose and multiple-ascending dose studies, no clinically meaningful changes were noted in hematology, chemistry, vital signs, or electrocardiogram parameters.

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Angela Genge, MD, FRCPC. [LISTEN TIME: 13 minutes]

Branded as Qalsody, the FDA has granted approval to Biogen and Ionis' tofersen, making it the fourth therapy approved for ALS and the first for SOD1-associated disease.

The director of the Massachusetts General Hospital ALS Care Center talked about CNM-Au8 treatment across three populations, as well as the HEALEY ALS Platform Trial, at the 2023 AAN annual meeting. [WATCH TIME: 5 minutes]

In a subgroup of patients with 300 m to 400 m 6MWD scores in the phase 3 Study 041, ataluren (Translarna; PTC Therapeutics) showed particular improvements from baseline.

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

The medical director of the Rocky Mountain Movement Disorders Center talked about the motivations of the phase 2 study on ANX005 for Huntington disease at the 2023 AAN Annual Meeting. [WATCH TIME: 3 minutes]

FDA-approved for adults with generalized myasthenia gravis in 2016, eculizumab continued to show promising efficacy and safety responses in adolescents with the disease, regardless of on concomitant use of IVIG.

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.