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Diroximel fumarate (DRF)(ALKS8700, BIIB098; Alkermes, Waltham, MA and Biogen, Cambridge, MA) is an oral fumarate in development for treatment of multiple sclerosis (MS). Like dimethyl fumarate (DMF) (Tecfidera; Biogen, Cambridge, MA), DRF is a prodrug that is converted to monomethyl fumarate. The unique chemical structure of DRF, however, creates potential for similar efficacy with better gastrointestinal tolerability; this is supported by interim data from week 48 of a 96-week open-label safety study (NCT02634307) presented at the American Academy of Neurology May 4-10 in Philadelphia,...
The Food and Drug Administration (FDA) has approved amifampridine (Ruzurgi; Jacobus Pharmaceutical Company, Princeton, NJ) for treating Lambert-Eaton myasthenic syndrome (LEMS) in patients age 6 to 17 years. This is the first FDA approval of a therapy specifically for pediatric patients with LEMS, a rare autoimmune neuromuscular junction disorder. Use of amifampridine in pediatric patients is supported by clinical studies in adults with LEMS, pharmacokinetic data in adult patients, pharmacokinetic modeling and simulation to identify the dosing regimen in pediatric...
Over 7,500 individuals have been or are being treated with nusinersen (Spinraza; Biogen, Cambridge, MA). Across clinical trials and the real-world setting, there are now 7 years of data and experience showing that nusinersen has clinical efficacy and safety. In the NURTURE study (NCT02386553) in which presymptomatic infants likely to develop spinal muscular atrophy type one (SMA1) or type 2 (SMA2) were treated with nusinersen, children continue to develop and gain typical motor milestones. After 26 months of treatment, all infants could sit without support; 88% could walk with support and 77%...
Niushen Zhang, MD
Cynthia E. Armand, MD; Alina Masters-Israilov, MD; and Richard B. Lipton, MD
Umer Najib, MD, FAHS; Jessica Frey, MD; and David B. Watson, MD, FAHS, FAAN