Neuromuscular

Barry J. Byrne, MD, PhD, the associate chair of pediatrics and director of the Powell Gene Therapy Center at the University of Florida; and the chief medical advisor to the Muscular Dystrophy Association, offered his perspective on the upcoming FDA decision on AT-GAA (Amicus Pharma).

Using patients with post–COVID-19 neurological symptoms, 90% of the cohort fulfilled the criteria for chronic fatigue syndrome, as expressed by the SOFA scale.

If approved, the oral fixed-dose coformulation of sodium phenylbutyrate-taurursodiol will become the third medication to receive an FDA greenlight for ALS.

The hold was originally placed on part B of the phase 2 MOMENTUM trial in Duchenne muscular dystrophy following a June 2022 report of hypomagnesemia after treatment with high-dose SRP-5051. Going forward, Sarepta will adjust its global trial protocol.

Olwen C. Murphy, MBBCh, and Matthew R. Vogt, MD, PhD, discussed the current clinical understanding of AFM and the need to recognize this condition amid the “peak” months of infection: August, September, and October.

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

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.

The director of medical outpatient services at the Kennedy Krieger Institute provided insight on how clinicians have typically assessed children who fail to meet milestones, and how that might change with new guidelines. [WATCH TIME: 3 minutes]

Compared with placebo, vamorolone-treated patients showed significantly better outcomes on several secondary and exploratory end points, such as the NorthStar Ambulatory Assessment and time to climb 4 stairs.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending September 2, 2022.

Using Strong et al’s classification criteria, the percentage of SOD1+ and SOD1- patients with altered cognitive and/or behavioral profiles were similar; however, cognitive and behavioral impairment differentiated between groups.

After switching from idursulfase to Denali’s DNL310, patients demonstrated mean declines of more than 80% for both heparan sulfate and dermatan sulfate biomarkers at week 49 of treatment.

The director of medical outpatient services at the Kennedy Krieger Institute discussed the changes and implications of new CDC guidelines on developmental milestones for growing children.

The director of medical outpatient services at the Kennedy Krieger Institute provided insight on the recently announced revisions to the “Learn the Signs. Act Early.” CDC developmental milestones for children. [WATCH TIME: 4 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 Parkinson disease and movement disorders.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending August 26, 2022.

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 sleep disorders.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending August 19, 2022.

Following positive phase 3 findings, a second study featuring more than 300 patients will aim to confirm PXT3003’s effect in patients with Charcot-Marie-Tooth disease type 1a.

The postdoctoral researcher at King’s College London provided background on why gene therapies have not seen as much success in treating ALS, and the need for additional validation of genetic mutations. [WATCH TIME: 3 minutes]

The correction in Muscle & Nerve resulted in a statistically significant treatment difference of more than 2 points for average change from baseline in ALSFRS-R in the prespecified efficacy subgroup of with a baseline score of at least 35.

The postdoctoral researcher at King’s College London provided insight on the immunologic differences between genes linked with ALS, and the important aspects to consider for gene-targeted therapies. [WATCH TIME: 3 minutes]