Isabella Ciccone, MPH

With the FDA approval, vamorolone (Agamree) becomes the first-in-class dissociative steroid therapy for patients with Duchenne muscular dystrophy. Catalyst Pharmaceuticals plans to launch the treatment in the first quarter of 2024.

If in-person interventions are unavailable, remote forms of telerehabilitation may be considered as a solution with very little cost-effectiveness and an easy option for physiotherapists and patients, even if it is for a short period of time.

Patients with mild Alzheimer disease who received high doses of BIIB080 demonstrated improvement in cognitive and functional outcomes compared with placebo, supporting further investigation.

Findings from the trial demonstrated rapid and sustained clinical response in patients with Alzhiemer disease agitation during the open-label treatment phase and did not show any new safety signals.

The data suggest that central nervous system-derived extracellular vesicles in the blood will likely play a key role in biomarker development, especially for patients with Parkinson disease, in the coming years.

Nancy Foldvary-Schaefer, DO, FAAN, director of the Sleep Disorders Center and staff in the Epilepsy Center at Cleveland Clinic, discussed the recent advancements in narcolepsy treatments and the importance of incorporating a holistic care approach for patients.

During the progression of the movement disorder, older patients with Parkinson disease may see changes in their insulin-like growth factor and serum homocysteine indices but a potential therapy could improve these factors.

The FDA’s decision to allow at-home dosing of intransal foralumab for patients with multiple sclerosis is likely to improve patient compliance to treatment and health outcomes, according to a recent release statement.

During the open-label treatment period, 83% of patients with idiopathic hypersomnia who completed treatment saw a significant reduction in sleepiness, with an average Epworth Sleepiness Scale change of -9.4 points.

Mark I. Boulos, MD, BSc, FRCP, CSCN, MSc, associate professor of medicine, division of neurology, Institute of Medical Science, University of Toronto, talked about a comprehensive meta-analysis aimed to determine normal mean sleep latency values to accurately help clinicians with interpreting sleep test results.

In a recent clinical trial, patients who received SaiLuoTong showed improvements in aspects of memory and executive function including delayed episodic memory retrieval, switching between cognitive concepts, higher-level divided attention, and multitasking compared with placebo.

Findings from the OCARINA I study presented at the MSMilan 2023 meeting showed that a 920 mg subcutaneous dose of ocrelizumab was well-tolerated in patients with relapsing or primary progressive multiple sclerosis, with similar exposure to the FDA-approved intravenous dose.

A recent analysis presented at MSMilan 2023 showed significant cognitive and biomarker improvements among patients with progressive multiple sclerosis receiving repeated intrathecal injections of autologous mesenchymal stem cells.

A new analysis of the SAkuraMoon study showed that annual relapse rate remained consistently low in satralizumab-treated patients, with high proportions of patients remaining free from relapse, severe relapse, and worsening in disability.

A recent analysis of the PREVENT and CHAMPION-NMOSD found no significant differences in safety outcomes for patients with AQP4+ NMOSD who were treated with rituximab prior to starting C5 complement inhibitors in specific timeframes.

Jacqueline French, MD, professor of neurology at NYU Grossman School of Medicine and chief medical officer of the Epilepsy Foundation, talked about findings from a newly published study assessing XEN1101 in patients with drug-resistant epilepsy.

Catch up on any of the neurology news headlines you may have missed over the course of September 2023, compiled all into one place by the NeurologyLive® team.

A recently developed machine learning model could analyze retinal images as well as associated data of specific features to recognize patients with mild cognitive impairment.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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