Send to

Choose Destination
See comment in PubMed Commons below
Neurology. 2007 Jan 2;68(1):51-5. Epub 2006 Nov 2.

Randomized, double-blind, placebo-controlled trial of phenylbutyrate in spinal muscular atrophy.

Author information

  • 1Department of Paediatric Neurology, Catholic University, Largo Gemelli, 00168 Rome, Italy.



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.


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.


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.


Phenylbutyrate was not effective at the regimen, schedule, and duration used in this study.

[PubMed - indexed for MEDLINE]
PubMed Commons home

PubMed Commons

How to join PubMed Commons

    Supplemental Content

    Full text links

    Icon for HighWire
    Loading ...
    Support Center