title: SMA valiant trial: a prospective, double-blind, placebo-controlled trial of valproic acid in ambulatory adults with spinal muscular atrophy.
abstract: BACKGROUND
  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.
  
  
  METHODS
  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, 6, and 12 months. The primary outcome was the 6-month change in maximum voluntary isometric contraction testing with pulmonary, electrophysiological, and functional secondary outcomes.
  
  
  RESULTS
  Thirty subjects completed the study. VPA was well tolerated, and compliance was good. There was no change in primary or secondary outcomes at 6 or 12 months.
  
  
  CONCLUSIONS
  VPA did not improve strength or function in SMA adults. The outcomes used are feasible and reliable and can be employed in future trials in SMA adults.
authors.forename: John T
authors.initials: JT
authors.lastname: Kissel
authors.forename: Bakri
authors.initials: B
authors.lastname: Elsheikh
authors.forename: Wendy M
authors.initials: WM
authors.lastname: King
authors.forename: Miriam
authors.initials: M
authors.lastname: Freimer
authors.forename: Charles B
authors.initials: CB
authors.lastname: Scott
authors.forename: Stephen J
authors.initials: SJ
authors.lastname: Kolb
authors.forename: Sandra P
authors.initials: SP
authors.lastname: Reyna
authors.forename: Thomas O
authors.initials: TO
authors.lastname: Crawford
authors.forename: Louise R
authors.initials: LR
authors.lastname: Simard
authors.forename: Kristin J
authors.initials: KJ
authors.lastname: Krosschell
authors.forename: Gyula
authors.initials: G
authors.lastname: Acsadi
authors.forename: Mary K
authors.initials: MK
authors.lastname: Schroth
authors.forename: Guy
authors.initials: G
authors.lastname: D'Anjou
authors.forename: Bernard
authors.initials: B
authors.lastname: LaSalle
authors.forename: Thomas W
authors.initials: TW
authors.lastname: Prior
authors.forename: Susan
authors.initials: S
authors.lastname: Sorenson
authors.forename: Jo Anne
authors.initials: JA
authors.lastname: Maczulski
authors.forename: Kathryn J
authors.initials: KJ
authors.lastname: Swoboda
mesh_terms: D000328:Adult; D000553:Ambulatory Care; D015331:Cohort Studies; D018592:Cross-Over Studies; D004305:Dose-Response Relationship, Drug; D004311:Double-Blind Method; D005260:Female; D056572:Histone Deacetylase Inhibitors / Q000494:pharmacology / Q000627:therapeutic use*; D006801:Humans; D008297:Male; D008875:Middle Aged; D009119:Muscle Contraction / Q000187:drug effects / Q000502:physiology; D053580:Muscle Strength / Q000187:drug effects / Q000502:physiology; D009134:Muscular Atrophy, Spinal / Q000188:drug therapy* / Q000503:physiopathology*; D011446:Prospective Studies; D016896:Treatment Outcome; D014635:Valproic Acid / Q000494:pharmacology / Q000627:therapeutic use*
chemical_list: D056572:Histone Deacetylase Inhibitors; D014635:Valproic Acid