Early Phase 1 Trial Data Suggests Therapeutic Potential for PGN-EDODM1 in Myotonic Dystrophy Type 1
February 25th 2025Initial data reported from the FREEDOM-DM1 trial suggest PGN-EDODM1 has dose-dependent effects in patients with DM1, with further results from study cohorts expected in 2025 and 2026.
Notable Sessions to Eye Amid the 2025 ACTRIMS Forum: Véronique Miron, PhD
February 25th 2025The John David Eaton Chair in Multiple Sclerosis at the University of Toronto gave clinical insights on emerging, innovative tracks at the 2025 ACTRIMS Forum and how these sessions align with recent trends in the multiple sclerosis field. [WATCH TIME: 4 minutes]
Migraine Medication Symbravo Demonstrates Therapeutic Efficacy in Patients Unresponsive to CGRPs
February 24th 2025Symbravo showed a statistically significant greater migraine treatment response compared to prior oral CGRP inhibitors (P <0.001), with 47.9% of patients reporting 2-hour pain freedom for most attacks versus 1.0% with oral CGRPs
Steps in Translating Genetic Discoveries Into Effective Treatments for ALS: Matthew B. Harms, MD
February 24th 2025The associate professor of neurology at Columbia University provided commentary on the rapid advancements in translating genetic research into ALS treatments and the challenges that remain. [WATCH TIME: 5 minutes]
FAERS Data Analysis Reveals Insights on Safety Profiles of Narcolepsy Treatments
February 23rd 2025A recent analysis identified potential safety concerns associated with pitolisant, sodium oxybate, solriamfetol, and modafinil, highlighting distinct adverse event profiles for each narcolepsy treatment.
Nuances in Thrombectomy Techniques and Anesthesia Use for Stroke: J Mocco, MD, MS
February 23rd 2025The director of the Cerebrovascular Center at Mount Sinai discussed how results from the ESCAPE-MeVO trial highlight the impact of anesthesia rates, thrombectomy techniques, and the need for rigorous clinical trials to minimize bias. [WATCH TIME: 3 minutes]
Hypertension Risk Elevated in Normotensive Patients With Narcolepsy Starting Sodium Oxybate
February 22nd 2025Normotensive patients with narcolepsy on sodium oxybate had over 50% of a higher risk of new-onset hypertension or antihypertensive medication use compared with nonusers of the treatment.
High-Risk Genes for Familial Hemiplegic Migraine Do Not Elevate Epilepsy Risk, Study Shows
February 21st 2025Pathogenic variants in the CACNA1A, ATP1A2, and SCN1A genes were associated with a higher risk of migraine, particularly in heterozygous carriers of loss-of-function and neutral variants.
The Rationale Behind Recently Approved AXS-07 Combination Therapy for Migraine: Stewart Tepper, MD
February 21st 2025The professor of neurology at the Geisel School of Medicine at Dartmouth talked about how the combination therapy involving meloxicam and rizatriptan may provide a more effective approach to treating migraine. [WATCH TIME: 6 minutes]
Oligoclonal Bands in Cerebrospinal Fluid May Signal Higher Relapse Risk in MOGAD
February 20th 2025A recent study reported that the presence of oligoclonal bands in cerebrospinal fluid of patients with myelin oligodendrocyte glycoprotein antibody-associated disease may be associated with a higher risk of relapse.
Navigating the Future of Gene Therapy in Neuromuscular Disorders: John Brandsema, MD
February 20th 2025The pediatric neurologist at Children’s Hospital of Philadelphia provided background on a notable track session at the upcoming MDA Clinical & Scientific Conference covering updates in gene therapy for neuromuscular conditions. [WATCH TIME: 4 minutes]
Managing NMOSD Disease Progression Through Healthy Habits and Holistic Care: Tammy Smith, MD, PhD
February 20th 2025The assistant professor in the neurology department at the University of Utah in Salt Lake City discussed the importance of addressing broader quality-of-life measures in patients with NMOSD beyond just preventing relapses. [WATCH TIME: 3 minutes]
FDA Refuses to File Harmony’s Supplemental NDA of Pitolisant in Idiopathic Hypersomnia
February 20th 2025Marketed as Wakix, the treatment is currently approved in the United States for excessive daytime sleepiness (EDS) or cataplexy in adults with narcolepsy and for EDS in pediatric patients aged 6 and older.