Years of genetic research has uncovered more about the underpinnings of limb-girdle muscular dystrophy, leading to potential disease-modifying therapies in the near future.

In honor of Limb-Girdle Muscular Dystrophy Awareness Day, held September 30, 2023, get caught up on some of the latest news in LGMD, with data updates and expert insights all in one place from the NeurologyLive® team.

The AI-based SOMNUM technology uses diagnostic algorithms based on multi-channel/time series sleep biosignal data to deliver fast, accurate diagnostic information for providers.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending September 29, 2023.

In a phase 3 setting, the 2-component therapy showed positive trends or clinically meaningful improvements on motor and respiratory functions compared with previously approved enzyme replacement therapy.

Erin Longbrake, MD, PhD, associate professor of neurology at Yale School of Medicine, discussed the importance of understanding rare autoimmune disorders to effectively design clinical trials and treatment strategies, especially for heterogeneous conditions like NMOSD and MOGAD.

Over a 4-week treatment period, patients in both Lu AG09222 groups experienced about 2 fewer migraine days per month compared with placebo.

As part of our monthly clinician spotlight, NeurologyLive® highlighted stroke expert Peter Kang, MD, director of the Paul and Sheila Wellstone Muscular Dystrophy Center and professor of neurology at the University of Minnesota.

Patients who self-identified as Black and were living in less affluent neighborhoods were less likely to be seen in one large memory clinic as well as have more severe dementia at initial visit.

After deliberating, the FDA committee expressed concerns about the mechanism of action of NurOwn, manufacturing details, and consistency of data.

By targeting tau-ack280, ADEL-Y01 inhibits tau aggregation and propagation, and promotes microglial tau clearance.

The phase 3 SYMPHONY trial assessing AXS-12, a selective norepinephrine reuptake inhibitor for narcolepsy, was initially expected in the third quarter of 2023.

The chief of the Movement Disorders Division at Mass General Hospital provided context on the goals behind the research careers reimagined course at the recently concluded ANA Annual Meeting.

Coya Therapeutics expects to release data from the biomarker analyses of COYA 302 and publish the full results from the trial in a peer-reviewed journal in the second half of the year.

Although research supports the use of physical therapy as an effective treatment for Parkinson disease, implementation factors including type, timing, frequency, and durability of outcomes remain mainly untouched.

Between ages 65 to 69, gene carriers of APOE ε4 could detect an average of about 3.2 of the smells presented, compared with about 3.9 smells for noncarriers of the gene variant.

Additional biomarker findings from up to 2 years of DNL310 treatment suggest positive changes in adaptive behavior and cognition as well as improvement in auditory function among pediatric patients with Hunter syndrome.

In an integrated meta-analysis of the HEALEY ALS platform trial and RESCUE-ALS trial, 30mg of CNM-Au8 resulted in 59% decreased risk of mortality among participants with ALS compared with PRO-ACT matched placebo over long-term follow-up.

Virginia Gao, MD, PhD, a movement disorders fellow at Weill Cornell Medicine, discussed several topics related to the use of biomarkers in the prodromal stages of neurodegenerative disorders like Parkinson disease.

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

A recent analysis showed a form of tau that could serve as a biomarker to track and explore whether investigational tau-based drugs are effective against Alzheimer disease.

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.

In the largest meta-analysis of patients with poststroke seizures, findings showed that those with early seizures had a greater mortality risk than those with late.

Among 443 episodes in 149 patients treated with different doses of BXCL501 over 12 weeks, the 60 mcg dose showed a reduction in agitation for the first and all treated episodes at 1 and 2 hours.

In the study, patients with ALS will be asked to complete questionnaires, a physical examination, and undergo neurostimulation site visits 3 times per week for 4 weeks.