Showing 21-30 of 3009 results for "".
- Evrysdi Therapy for Type 1 Spinal Muscular Atrophy Associated with Favorable 5-Year S&E Datahttps://practicalneurology.com/news/evrysdi-therapy-for-type-1-spinal-muscular-atrophy-associated-with-favorable-5-year-se-data/2470501/Five-year data from the open-label extension of the phase 2 FIREFISH (NCT02913482) clinical trial confirms the sustained efficacy and safety profile of Evrysdi (ridisplam; Genentech, South San Francisco, CA), a treatment for children with Type 1 spinal muscular atrophy (SMA). The results demonstr
- Subcutaneous Lecanemab Shown to Have Similar Efficacy to IV Lecanemab and May Reduce ARIA-Ehttps://practicalneurology.com/news/subcutaneous-lecanemab-shown-to-have-similar-efficacy-to-iv-lecanemab-and-may-reduce-aria-e/2470249/Subcutaneous injection of lecanemab (Eisai, Tokyo, Japan; Biogen, Cambridge, MA) using a vial or autoinjector yielded comparable average steady-state concentrations (Css) to intravenous (IV) administration o
- Possible Treatment for Focal Laryngeal Dystonia Revealed at MDS Conferencehttps://practicalneurology.com/news/possible-treatment-for-focal-laryngeal-dystonia-reported-at-mds-conference/2470597/Results of a double-blind, placebo-controlled, randomized cross-over study presented at the 2024 International Congress of Parkinson’s Disease and Movement Disorders suggest that sodium oxybate (SO) may be an effective treatment for some people with focal laryngeal dystonia (LD). This phase
- In Memoriam: Ralph L. Sacco, MD, MS, FAHA, FAANhttps://practicalneurology.com/news/in-memoriam-ralph-l-sacco-md-ms-faha-faan/2470102/
- Ludwig Kappos, MD, Awarded 2026 John Dystel Prize for MS Research, to Present at AAN 2026https://practicalneurology.com/news/ludwig-kappos-md-awarded-2026-john-dystel-prize-for-ms-research-to-present-at-aan-2026/2486639/
- Pitolisant Clinical Trial for Type 1 Myotonic Dystrophy Excessive Daytime Sleepinesshttps://practicalneurology.com/news/pitolisant-clinical-trial-for-type-1-myotonic-dystrophy-excessive-daytime-sleepiness/2469633/A phase 2 clinical trial (NCT04886518) for pitolisant (Wakix; Harmony Biosciences, Plymouth Meeting, PA) in adults with type 1 myotonic dystrophy (DM1) experiencing excessive daytime sleepiness (E
- Azeliragon Studies for Treatment of Alzheimer Disease In People With Type II Diabetes Continuehttps://practicalneurology.com/news/azeliragon-studies-for-treatment-of-alzheimer-disease-in-people-with-type-ii-diabetes-continue/2469421/Baseline characteristics for participants enrolled in a phase 2/3 study, Elevage (NCT03980730), of azeliragon (vTv Therapeutics, High Point, NC) for mild Alzheimer disease (AD) in people with type 2 diabetes have been reported. In an e
- Vatiquinone Proceeds to Phase 2/3 Studies for Treatment of Mitochondrial Epilepsyhttps://practicalneurology.com/news/vatiquinone-proceeds-to-phase-23-studies-for-treatment-of-mitochondrial-epilepsy/2469409/A registration-directed phase 2/3 clinical trial (NCT04378075) to evaluate vatiquinone (PTC743; PTC Therapeutics, South Plainfield, NJ) in individuals with mitochondrial epilepsy (MIT-E) has begun
- Barancik Prize Awarded to Dr. Ruth Ann Marrie at ACTRIMS 2023https://practicalneurology.com/news/barancik-prize-awarded-to-dr-ruth-ann-marrie-at-actrims-2023/2470120/The National Multiple Sclerosis Society announced that Ruth Ann Marrie, MD, P
- Fenfluramine May Usher in Higher Standards for Treatment of Dravet Syndromehttps://practicalneurology.com/news/fenfluramine-may-usher-in-higher-standards-for-treatment-of-dravet-syndrome/2469642/In an editorial in Epilepsy and Behavior, Helen Cross, MD and Joseph Sullivan, MD suggest fenfluramine (Fintepla; Zogenix, Emeryville, CA) clinical trial results can raise the bar for efficacy of antiseizure medications (ASMs) for