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The Food and Drug Administration (FDA) has approved onasemnogene abeparvovac-xioi (Zolgensma; Avexis, Bannockburn, IL) for the treatment of all types of spinal muscular atrophy (SMA) in children under age 2 years. Approved indications for onasemnogene, an SMN gene-replacement therapy, include all types of SMA. Treatment of presymptomatic children diagnosed through genetic screening is also approved. Not all states include testing for SMA in newborn screening panels, however. Onasemnogene is given intravenously in a single 1-time infusion. In 2 clinical trials (STRIVE [NCT03306277] and...
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...
In a double-blind phase 2 study (NCT02975349), adult participants with multiple sclerosis (MS) who were treated with evobrutinib (EMD-Serono, Rockland, MA) (75 mg taken orally once or twice daily) had reduced numbers of T1 gadolinium enhancing (Gd+) lesions as early as 12 weeks after initiating treatment (Table 1). This reduction in T1 Gd+ lesions was maintained through week 48 of the study (Table 2). Participants treated with evobrutinib also had reduced annualized relapse rate (ARR) compared with those treated with placebo (Table 1), although this did not reach statistical...
Saif A. Bushnaq, MD; and Sunil A. Sheth, MD
Umer Najib, MD, FAHS; Jessica Frey, MD; and David B. Watson, MD, FAHS, FAAN
Niushen Zhang, MD