Showing 281-290 of 1728 results for "".
- FDA Expands Indication for Brineura To Treat CLN2 Diseasehttps://practicalneurology.com/news/fda-expands-indication-for-brineura-to-treat-cln2-disease/2470546/The Food and Drug Administration (FDA) has approved the supplemental Biologics License Application (sBLA) for Brineura (cerliponase alfa; BioMarin Pharmaceuticals, San Rafael, CA), expanding the medication’s indication to be used as a treatment to slow the loss of ambulation in all children
- Durable Benefit from Treating Batten's Disease with Cerliponase Alfahttps://practicalneurology.com/news/durable-benefit-from-treating-battons-disease-with-cerliponase-alfa/2468837/In an ongoing, open-label extension study of cerliponase alfa (Brineura; BioMarin, San Rafael, CA) for treatment of patients with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as Batten's disease, the rate of clinical decline continued to decrease over
- 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/
- 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
- Award Recipients Recognized at the 2024 International Congress of Parkinson’s Disease and Movement Disordershttps://practicalneurology.com/news/award-recipients-recognized-at-the-2024-international-congress-of-parkinsons-disease-and-movement-disorders/2470598/The International Parkinson’s Disease and Movement Disorders Society (MDS) has announced recipients of the 2024 MDS Awards. The awards honor professionals in the field who are elevating care for movement disorders through their contributions to research, scholarship, and clinical work. The
- FDA Approves Mirabegron for Neurogenic Detrusor Overactivity in Pediatric Patientshttps://practicalneurology.com/news/fda-approves-mirabegron-for-neurogenic-detrusor-overactivity-in-pediatric-patients/2469543/The Food and Drug Administration approved a new indication for mirabegron extended-release tablets (Myrbetriq; Silver Spring, MD) and mirabegron for extended-release oral suspension (Myrbetriq Granules; Silver Spring, MD) to treat neurogenic detrusor overactivity (NDO) in children age 3 yea
- Vamorolone Has Efficacy for Treating Duchenne Muscular Dystrophy Without Affecting Growthhttps://practicalneurology.com/news/vamorolone-has-efficacy-for-treating-duchenne-muscular-dystrophy-without-affecting-growth/2470013/In the VISION-DMD trial (NCT02760277), children with Duchenne muscular dystrophy (DMD) treated with vamorolone (ReveraGen BioPharma, Rockville, MD), had improved motor function with no negative effects on linear growth or biomarkers of
- Sargramostim Improves Motor Symptoms of Parkinson Disease in Small Phase 1 Trialhttps://practicalneurology.com/news/sargramostim-improves-motor-symptoms-of-parkinson-disease-in-small-phase-1-trial/2469597/An investigator-initiated clinical trial (NCT03790670) evaluated the use of sargramostim (Leukine; Partner Therapeutics, Lexington, MA). Improvements in MDS-UPDRS Part 3 scores, which measure motor sympto