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From the Department of Paediatric Neurology, Catholic University, Rome, Italy (E.M., F.C., M. Pane, M. Pelliccioni); Dubowitz Neuromuscular Centre, Hammersmith Hospital, Imperial College, London, UK (E.M., M.K.); Department of Laboratory Medicine, Unit of Molecular Medicine, Bambino Gesù Hospital, Rome, Italy (E.B., A.D.); Department of Neurosciences, Psychiatry and Anaesthesiology, University of Messina, Italy (S.M., G.V.); Division of Neuroepidemiology, National Neurological Institute C. Besta, Milan, Italy (A.S.); Institute of Medical Genetics, Catholic University, Rome, Italy (C.A., G.N., F.D.T., C.B.); Department of Developmental Neuroscience, IRCCS Stella Maris, Pisa, Italy (R.B., C.C.); Department of Child Neurology and Psychiatry, IRCCS "C. Mondino" Foundation, University of Pavia, Italy (A.B., S.O.); Department of Child Neuropsychiatry, University of Turin, Italy (P.B.); Neuromuscular Disease Unit, G. Gaslini Institute, Genoa, Italy (C.B., C.M.); Neuromuscular Center, S.G. Battista Hospital, University of Turin, Italy (T.M.); Myopathology and Immunology Unit, National Neurological Institute C. Besta, Milan, Italy (L.M.); UILDM, Rome, Italy (M. Pelliccioni); Child Neurology and Psychiatry Unit, Maggiore Hospital, Bologna, Italy (A.P.); and Nigrisoli Hospital, Bologna, Italy (M.V.).
* To whom correspondence should be addressed. E-mail: e.mercuri{at}imperial.ac.uk.
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.
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