Considerations and Opportunities With Approval of Eplontersen for Hereditary ATTR Polyneuropathy
December 22nd 2023Sami 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.
FDA Approves Eplontersen for Polyneuropathy of Hereditary Transthyretin Amyloidosis
December 22nd 2023With the approval, eplontersen becomes the first approved treatment for patients with hereditary transthyretin-mediated amyloid polyneuropathy that can be self-administered via an autoinjector.
Rett Syndrome Symptoms and Quality of Life Improvements Through Trofinetide: Ponni Subbiah, MD, MPH
December 21st 2023The 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]
Management of Focal Onset Seizures With Brivaracetam Adjunctive Therapy: Iryna Leunikava, MSc
December 21st 2023The 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]
Accurate Diagnosis of Functional Motor Disorders in Elders Critical to Long-Term Care, Management
December 21st 2023The 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.
Gene Therapy AMT-130 Shows Potential Benefit in Phase 1/2 Clinical Trials for Huntington Disease
December 20th 2023In 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.
NeurologyLive® Clinician of the Month Spotlight: Amaal J. Starling, MD, FAHS, FAAN
December 20th 2023As 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.
IJMSC Insights: MS and Mobility With Laura Rice
December 20th 2023A 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).
NeuroVoices: Ponni Subbiah, MD, MPH, on the Open-Label LILAC-2 Study of Trofinetide
December 20th 2023The 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.
Study Reveals Core Recommendations to Improve Palliative Care in Parkinson Disease
December 19th 2023A 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.
Case Study Reveals Novel Intragenic Deletion of the FXN Gene in Friedreich Ataxia
December 19th 2023In 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.
Quantifying Impact of Tuberous Sclerosis Complex-Associated Neuropsychiatric Disorders
December 19th 2023Elizabeth 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.
Parkinson Disease Symptoms Improved With Modification of Advanced Therapies
December 18th 2023A 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.
Understanding Stathmin-2’s Impact on ALS Motor and Sensory Denervation: Cathleen M. Lutz, PhD
December 18th 2023The 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]