Neuromuscular

After 1 year of treatment with risdiplam, 81% of the 26-infant cohort were able to sit independently for 30 seconds, a milestone not typically reached in SMA.

Neurology News Network for the week ending October 7, 2023. [WATCH TIME: 3 minutes]

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending October 6, 2023.

RGX-202, a gene therapy for Duchenne muscular dystrophy, was well-tolerated with no therapy-related serious adverse effects in 3 patients who received the level 1 dosage.

The chief scientific officer of AMO Pharma discussed results from a recent clinical trial evaluating the safety and efficacy impact of AMO-02 on children and adolescents with congenital myotonic dystrophy. [WATCH TIME: 6 minutes]

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Gunter Hoglinger, MD. [LISTEN TIME: 14 minutes]

The phase 3 study is expected to include 86 children with ataxia-telangiectasia who will be assessed on changes in a rescored modified International Cooperative Ataxia Rating Scale.

The chief medical advisor at the Muscular Dystrophy Association gave his reaction to Amicus Pharmaceuticals’ recently FDA approved 2-part therapy for patients living with Pompe disease. [WATCH TIME: 7 minutes]

After being diagnosed with acute flaccid myelitis more than 10 years ago, patient advocate Sarah Todd Hammer spoke on the ways in which disability should be viewed and discussed.

In a cross-study analysis, omaveloxolone slowed progression in FA by at least 50% each year for 3 years compared with corresponding data from an external control group in a natural history study.

Previous data on the 25 mg cohort of CTI-1601 showed significant reductions in frataxin levels among treated individuals with Friedreich ataxia.

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

Over a 4-year follow-up, SARA progression was substantially linear. Age at evaluation also influenced the speed of SARA progression, while disease duration did not improve the prediction of the statistical model.

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.

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.

Neurology News Network for the week ending September 30, 2023. [WATCH TIME: 4 minutes]

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

The professor of neurology and translational neuroscientist at Ludwig-Maximillian’s University Munich detailed the logic behind the ORION study, a new trial assessing AMX0035, an approved therapy for ALS, in patients with progressive supranuclear palsy. [WATCH TIME: 4 minutes]

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.

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

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

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.

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.