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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%...
The American Stroke Association (ASA) has published updated recommendations for stroke care systems (Stroke. 2019;50:00-00). The recommendations comprehensively review evidence evaluating stroke care systems and update previous ASA recommendations based upon advances in stroke care systems. Among such advances are stroke center certifications, remarkable improvement in endovascular therapy, neurocritical care, telestroke, mobile stroke units, and other innovations. All of this has occurred while the landscape of health care in the US has undergone large changes. The statement addresses...
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,...
Niushen Zhang, MD
Brian E. McGeeney, MD, MPH, MBA
Emmanuelle A. D. Schindler, MD, PhD; and Christopher Gottschalk, MD