Ontology highlight
ABSTRACT:
SUBMITTER: Kissel JT
PROVIDER: S-EPMC3888833 | biostudies-literature | 2014 Feb
REPOSITORIES: biostudies-literature
Kissel John T JT Elsheikh Bakri B King Wendy M WM Freimer Miriam M Scott Charles B CB Kolb Stephen J SJ Reyna Sandra P SP Crawford Thomas O TO Simard Louise R LR Krosschell Kristin J KJ Acsadi Gyula G Schroth Mary K MK D'Anjou Guy G LaSalle Bernard B Prior Thomas W TW Sorenson Susan S Maczulski Jo Anne JA Swoboda Kathryn J KJ
Muscle & nerve 20140201 2
<h4>Introduction</h4>An open-label trial suggested that valproic acid (VPA) improved strength in adults with spinal muscular atrophy (SMA). We report a 12-month, double-blind, cross-over study of VPA in ambulatory SMA adults.<h4>Methods</h4>There were 33 subjects, aged 20–55 years, included in this investigation. After baseline assessment, subjects were randomized to receive VPA (10–20 mg/kg/day) or placebo. At 6 months, patients were switched to the other group. Assessments were performed at 3, ...[more]