Sami Khella, MD, professor of clinical neurology at the Perelman School of Medicine at the University of Pennsylvania School of Medicine, shared his reaction to the approval of eplontersen for the treatment of hereditary transthyretin-mediated amyloid polyneuropathy.
Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending December 22, 2023.
A new study revealed daytime continuous polysomnography as a reliable and easily accessible diagnostic tool for identifying narcolepsy type 1 among pediatric patients.
With the approval, eplontersen becomes the first approved treatment for patients with hereditary transthyretin-mediated amyloid polyneuropathy that can be self-administered via an autoinjector.
Researchers have successfully grown norepinephrine neurons from stem cells, which are critical for regulating various bodily functions, offering new insights into neurodegenerative diseases such as Alzheimer and Parkinson.
Click through for the most-read articles from the 2023 issues of the International Journal of MS Care: cannabis, fall prevention, parental MS, and more.
The chief medical officer of Acadia Pharmaceuticals provided commentary on a caregiver analysis assessing beneficial experiences with trofinetide in patients with Rett syndrome. [WATCH TIME: 4 minutes]
The European evidence generation strategy lead for epilepsy and rare syndromes at UCB Pharma talked about positive results from observational studies assessing the impact of brivaracetam among patients with focal seizures, which was presented at AES 2023. [WATCH TIME: 5 minutes]
The research aimed to assess FMD prevalence and clinical manifestations in both elderly and younger onset individuals, revealing insights into symptom variations across different age groups.
The newly announced trial, dubbed ReTain, will featured 500 individuals with preclinical Alzheimer disease who will be randomized to ACI-35.030 or placebo for a maximum of 4 years.
According to a recent study, investigators found no reports of moderate or severe adverse events with a high dose of nicotinamide riboside therapy in patients with Parkinson disease.
In newly released data, AMT-130 showed a manageable safety profile at both low and high doses in patients with Huntington disease, supporting the gene therapy's continued development.
As part of our monthly clinician spotlight, NeurologyLive® highlighted headache medicine expert Amaal J. Starling, MD, FAHS, FAAN, associate professor in the department of neurology at Mayo Clinic College of Medicine.
A new feature on NeurologyLive®, IJMSC Insights offers a closer look at the latest research and the people behind it from the community of the International Journal of Multiple Sclerosis Care (IJMSC) and the Consortium of Multiple Sclerosis Centers (CMSC).
The senior vice president, global head of medical affairs, and chief medical officer at Acadia Pharmaceutical, gave an overview of LILAC-2, a long-term extension study assessing trofinetide (Daybue), the first approved therapy for Rett syndrome.
A recent systematic practice review outlined pragmatic recommendations to enhance palliative care for patients with Parkinson disease and their caregivers, emphasizing integration, coordination, and personalized care plans.
In a recent case study of a 32-year-old man diagnosed with Friedreich ataxia, parental sample testing identified a novel intragenic deletion involving the 5'UTR upstream region and exons 1 and 2 of the FXN gene.
Elizabeth M. Cassidy, MPH, research project manager at the TSC Alliance, provided clarity on a presentation at AES 2023 examining the association between TSC-associated neuropsychiatric disorders and seizure duration.
The senior vice president of medical and clinical affairs for Avadel Pharmaceuticals provided commentary on recently published research supporting once-nightly sodium oxybate (Lumryz) in narcolepsy regardless of the subtype. [WATCH TIME: 5 minutes]
In a phase 3 study, patients on omaveloxolone had less physical impairment compared with patients who received placebo, as measured by the modified Friedreich Ataxia Rating Scale.
A new retrospective analysis revealed that modifying advanced therapies led to improved motor and subjective symptoms, emphasizing the importance of considering combination therapies when efficacy declines or adverse events arise.
The vice president of the Rare Disease Translational Center at the Jackson Laboratory discussed results from a recently published study reinforcing stathmin-2 as a potential therapeutic strategy for ALS. [WATCH TIME: 4 minutes]
A-synuclein-positive athletes were similar to α-synuclein-negative athletes on demographics and other serum and blood biomarkers; however, these patients had lower grey matter volumes in the right inferior orbitofrontal, right anterior insula, and right olfactory cortices.
A new feature on NeurologyLive®, IJMSC Insights offers a closer look at the latest research and the people behind it from the community of the International Journal of Multiple Sclerosis Care (IJMSC) and the Consortium of Multiple Sclerosis Centers (CMSC).
Here's some of what is coming soon to NeurologyLive® this week.
A recent meta-analysis revealed significant differences in characteristic between patients with NMOSD and MS, highlighting the need for enhanced tools to differentiate between these diseases for early and accurate diagnosis.
The clinical professor of neurology at Stanford University School of Medicine talked about the collaboration of Rapport leveraging NeuroPace’s data analysis capabilities to study the target delivery of a therapy for focal epilepsy. [WATCH TIME: 5 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 ataxia.
A recent subgroup analysis of the N-MOmentum study revealed that inebilizumab had equal efficacy in reducing attacks among Asian and nonAsian patients with NMOSD, demonstrating its continued superiority over placebo.
Subgroups of narcolepsy types 1 and 2 had significant reductions in a number of secondary outcomes, including number of sleep stage transitions, number of nocturnal arousals, and improvements in sleep quality.
