Neuromuscular

Investigators concluded that those receiving avalglucosidase alfa should be tested for antidrug antibodies by physicians who treat Pompe disease and monitor antidrug antibody, according to a recent Sanofi investigation.

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Ambereen Mehta, MD, MPH. [LISTEN TIME: 40 minutes]

ADAPT+ study data on antiacetylcholine receptor antibody-negative patients with generalized myasthenia gravis found improvements in cycle baseline and efficacy with Efgartigimod.

The associate professor at the University of Michigan provided insight on new guidelines to treat painful diabetic neuropathy. [WATCH TIME: 2 minutes]

More than 85% of the individuals who continued givosiran (Givlaari; Alnylam) through the 36-week open-label extension were attack free in the final 3 months of treatment.

From the pre-COVID period to 6 weeks after vaccination, the reporting rate of GBS was significantly different, regardless of whether Brighton criteria was applied to the analysis. The authors noted that passive surveillance limitations warrant further analysis.

Results from phase 2 RESCUE-ALS trial show that treatment with CNM-Au8 in ALS demonstrated improvement in survival compared to the estimated median survival rate.

The phase 3 study, paused at US sites by the FDA, seeks to enroll 154 individuals with generalized myasthenia gravis, both seropositive and seronegative.

After 12 weeks of treatment with zilucoplan 0.3 mg/kg, almost three-fourths of patients demonstrated at least a 3-point reduction in Myasthenia Gravis Activities of Daily Living scores.

Most ambulatory patients had improved pulmonary functions and biomarker outcomes with cipaglucosidase alfa/miglustat that were sustained through a less than 36-month follow-up period.

After demonstrating a positive reduction of mutant huntingtin protein in preclinical studies, WVE-003 continued to show similar results when assessed in 30- and 60-mg doses.

A single, 500-mg intravenous dose of rituximab in individuals with new-onset generalized myasthenia gravis reduced the risk of disease manifestation and the need for rescue medications.

Study investigator Jeffrey L. Neul, MD, PhD, offered his perspective on the current understanding of trofinetide (Acadia Pharmaceuticals) and its potential in Rett syndrome if approved by the FDA in the coming months.

The clinical director of the National Institute of Neurological Disorders and Stroke provided context on two recent papers that highlight a reactivation of HERV-K virus in the development of amyotrophic lateral sclerosis. [WATCH TIME: 5 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 stroke and cerebrovascular disease.

The explosion of new science and legislative decisions have expanded the capabilities and reach of newborn screening, providing thousands of families every year an opportunity to seek early, and critical, care for their infants.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending September 16, 2022.

The director of medical outpatient services at the Kennedy Krieger Institute discussed the long-term effects of the updated developmental milestone guidelines and the need to judge children based on the 75th percentile. [WATCH TIME: 7 minutes]

The treatment, previously known as NNZ-2566, is supported by data from the LAVENDER study. With no plans to hold an advisory meeting, the agency set a PDUFA date of March 12, 2023, for the Acadia Pharmaceuticals product.

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

The director of medical outpatient services at the Kennedy Krieger Institute discussed how the clinical community has approached children who fail to reach developmental milestones in part because of poor home lifestyle. [WATCH TIME: 3 minutes]

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.

Neurology News Network for the week ending September 10, 2022. [WATCH TIME: 4 minutes]

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending September 9, 2022.

The investigational Ionis and AstraZeneca treatment showed clinically meaningful changes in serum TTR concentration and modified Neuropathy Impairment Score +7 measures. The companies intend to file an NDA based on the findings of the NEURO-TTRansform study.

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.