title: Randomized, double-blind, placebo-controlled trial of phenylbutyrate in spinal muscular atrophy.
abstract: OBJECTIVE
  To assess the efficacy of phenylbutyrate (PB) in patients with spinal muscular atrophy in a randomized, double-blind, placebo-controlled trial involving 10 Italian centers.
  
  
  METHODS
  One hundred seven children were assigned to receive PB (500 mg/kg/day) or matching placebo on an intermittent regimen (7 days on/7 days off) for 13 weeks. The Hammersmith functional motor scale (primary outcome measure), myometry, and forced vital capacity were assessed at baseline and at weeks 5 and 13.
  
  
  RESULTS
  Between January and September 2004, 107 patients aged 30 to 154 months were enrolled. PB was well tolerated, with only one child withdrawing because of adverse events. Mean improvement in functional score was 0.60 in the PB arm and 0.73 in placebo arm (p = 0.70). Changes in the secondary endpoints were also similar in the two study arms.
  
  
  CONCLUSIONS
  Phenylbutyrate was not effective at the regimen, schedule, and duration used in this study.
authors.affiliation: Department of Paediatric Neurology, Catholic University, Largo Gemelli, 00168 Rome, Italy. e.mercuri@imperial.ac.uk
authors.forename: E
authors.initials: E
authors.lastname: Mercuri
authors.forename: E
authors.initials: E
authors.lastname: Bertini
authors.forename: S
authors.initials: S
authors.lastname: Messina
authors.forename: A
authors.initials: A
authors.lastname: Solari
authors.forename: A
authors.initials: A
authors.lastname: D'Amico
authors.forename: C
authors.initials: C
authors.lastname: Angelozzi
authors.forename: R
authors.initials: R
authors.lastname: Battini
authors.forename: A
authors.initials: A
authors.lastname: Berardinelli
authors.forename: P
authors.initials: P
authors.lastname: Boffi
authors.forename: C
authors.initials: C
authors.lastname: Bruno
authors.forename: C
authors.initials: C
authors.lastname: Cini
authors.forename: F
authors.initials: F
authors.lastname: Colitto
authors.forename: M
authors.initials: M
authors.lastname: Kinali
authors.forename: C
authors.initials: C
authors.lastname: Minetti
authors.forename: T
authors.initials: T
authors.lastname: Mongini
authors.forename: L
authors.initials: L
authors.lastname: Morandi
authors.forename: G
authors.initials: G
authors.lastname: Neri
authors.forename: S
authors.initials: S
authors.lastname: Orcesi
authors.forename: M
authors.initials: M
authors.lastname: Pane
authors.forename: M
authors.initials: M
authors.lastname: Pelliccioni
authors.forename: A
authors.initials: A
authors.lastname: Pini
authors.forename: F D
authors.initials: FD
authors.lastname: Tiziano
authors.forename: M
authors.initials: M
authors.lastname: Villanova
authors.forename: G
authors.initials: G
authors.lastname: Vita
authors.forename: C
authors.initials: C
authors.lastname: Brahe
mesh_terms: D002648:Child; D002675:Child, Preschool; D004311:Double-Blind Method; D005260:Female; D006801:Humans; D008297:Male; D009134:Muscular Atrophy, Spinal / Q000188:drug therapy* / Q000453:epidemiology / Q000503:physiopathology; D010654:Phenylbutyrates / Q000627:therapeutic use*; D012189:Retrospective Studies
chemical_list: D010654:Phenylbutyrates; C075773:4-phenylbutyric acid