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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 codirector of interventional neuroradiology and vice-chief of the department of neuroscience at Baptist Health Miami Neuroscience Institute discussed the questions that still remain with identifying and treating subarachnoid hemorrhage. [WATCH TIME: 3 minutes]

During the week after emergency department discharge, both low- and high-dose participants reported a median of 2 headache days, signifying little difference.

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]

In a cohort of moderately severe Alzheimer disease, patients on placebo demonstrated cognitive decline while those on active treatment showed no significant decline.

Researchers proposed a harmonized definition of progression independent of relapse activity that may improve the comparability of results in current and future study cohorts, according to a newly published systematic review.

Using treatment failures and antiseizure medication retention, findings pointed to levetiracetam as a more effective initial monotherapy in females with juvenile myoclonic epilepsy.

Elena Koundourakis, PhD, head of orexin franchise development at Takeda, talked about the phase 2 trial investigating TAK-994 in patients with narcolepsy type 1, which showed significant improvements in wakefulness, despite discontinuation because of hepatotoxicity.

The clinical professor of neurology at Lenox Hill Hospital/Northwell Health talked about her experience with monoclonal antibodies for Alzheimer disease treatment as well as the challenges and risks associated with them. [WATCH TIME: 8 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.

At the conclusion of a 12-weel treatment period, biomarker data provided mechanistic insights of senolytic effects using dasatinib and quercetin that need to be confirmed in fully powered, placebo-controlled studies.

The associate professor of neurology at Icahn School of Medicine at Mount Sinai talked about the promising new treatments for multiple sclerosis, the significance of the spinal cord in the disease, and the use of artificial intelligence in neuroimaging. [WATCH TIME: 3 minutes]

A recent finding from a qualitative study raises concerns about patient safety in healthcare because the system in the US is not well equipped to address the unique needs of older adults living alone with cognitive decline.

The director of the MedStar Georgetown Headache Center discussed the importance of a healthy clinician-patient relationship and the conversations needed during treatment selection for migraine. [WATCH TIME: 4 minutes]

The adjusted odds ratios observed for any vaccine and MS flare-ups requiring hospitalization were similar regardless of sex, with the highest odds observed for the pneumococcal vaccine.

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.

Although the mortality among monitored and nonmonitored patients was similar, the monitored group had significantly more patients with severe disability and fewer with good recovery at 6 months.

The professor of neurology at the Geisel School of Medicine at Dartmouth talked about recent studies presented at the International Headache Congress on a potential migraine prevention treatment targeting pituitary adenylate cyclase activating polypeptide. [WATCH TIME: 5 minutes]

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

The codirector of interventional neuroradiology and vice-chief of the department of neuroscience at Baptist Health Miami Neuroscience Institute provided clarity on the complexities with treating subarachnoid hemorrhage on an individual case basis. [WATCH TIME: 4 minutes]

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.

The professor of neurology at NYU Langone Grossman School of Medicine discussed the Women in Neurology session she gave at IFN 2023, where women shared their experiences from their medical careers. [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 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.