Potassium Channel Opener XEN1101 Offers Simplicity and Strong Efficacy in Seizure Control
October 10th 2023Jacqueline 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.
Search for Targeted Therapies for Primary Headaches Beyond Migraine: Jihan A. Grant, MD
October 10th 2023The director of headache medicine at The Mount Sinai Hospital talked about the often-overlooked realm of primary headaches beyond migraines as well as the importance of developing targeted treatments and increasing understanding of their pathophysiology. [WATCH TIME: 7 minutes]
Dosing Underway for Phase 1 Parkinson Trial of GBA1-Targeting Agent GT-02287
October 9th 2023In preclinical models, treatment with GT-02287 restored GCase enzymatic function, reduced aggregated a-synuclein, neuroinflammation and neuronal death, increased dopamine levels, and improved motor function.
FDA Issues Complete Response Letter to Alnylam for Patisiran in ATTR Amyloidosis Cardiomyopathy
October 9th 2023In a phase 3 study, patisiran met its primary and first secondary end point, demonstrating significant benefit in functional capacity, quality of life, and health status, in comparison with placebo.
Role of Sleep Coaching, Sleep Reset in the Care of Sleep Problems
October 6th 2023Michael Grandner, PhD, associate professor of psychiatry at the University of Arizona College of Medicine, discussed Sleep Reset, an app-based, personalized sleep program that uses sleep coaches to help guide patients in the care of their sleep disorders.
Assessing Phase 2/3 Trial Safety and Efficacy of AMO-02 for Myotonic Dystrophy: Mike Snape, PhD
October 6th 2023The 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]
Future Research on Subarachnoid Hemorrhage: Guilherme Dabus, MD
October 5th 2023The 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]
Clinical Viewpoint on Newly Approved Combination Therapy for Pompe Disease: Barry J. Byrne, MD, PhD
October 5th 2023The 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]
PIRA Identified as the Most Common Form of Disability Accumulation in Multiple Sclerosis
October 4th 2023Researchers 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.
Novel Insights on Orexin Receptor TAK-994 and its Discontinued Narcolepsy Study
October 4th 2023Elena 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.
Diving Into the Potential of Monoclonal Antibodies for Alzheimer Disease Treatment: Gayatri Devi, MD
October 4th 2023The 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]
Combination Therapy Demonstrates Ability to Clear Senescent Cells in Alzheimer Disease
October 3rd 2023At 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.