id author title date pages extension mime words sentences flesch summary cache txt cord-320979-25ytt84j De Vivo, Darryl C. Nusinersen initiated in infants during the presymptomatic stage of spinal muscular atrophy: Interim efficacy and safety results from the Phase 2 NURTURE study 2019-09-12 .txt text/plain 7626 327 49 Secondary endpoints reported in this interim analysis include (1) proportion of participants alive; (2) attainment of motor milestones as assessed by World Health Organization (WHO) criteria; (3) attainment of motor milestones by Hammersmith Infant Neurologic Examination, Section 2 (HINE-2); (4) change from baseline in the Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) motor function scale; (5) change from baseline in growth parameters: weight for age/length, head circumference, chest circumference, head-to-chest circumference ratio, and arm circumference; and (6) proportion of participants developing clinically manifested SMA at 13 and 24 months of age as defined by any of the following conditions: (a) age-adjusted weight < 5th percentile or decrease of ≥2 major weight growth curve percentiles (3rd, 5th, 10th, 25th, or 50th) compared with baseline, or a percutaneous gastric tube placement for nutritional support at 13 or 24 months of age; (b) failure to achieve sitting without support, standing with assistance, and hands-and-knees crawling at age 13 months; or (c) failure to achieve the milestones defined at age 13 months and failure to achieve walking with assistance, standing alone, and walking alone at age 24 months. ./cache/cord-320979-25ytt84j.txt ./txt/cord-320979-25ytt84j.txt