Neurology
HOME HELP FEEDBACK SUBSCRIPTIONS ARCHIVE SEARCH TABLE OF CONTENTS
QUICK SEARCH:   [advanced]


     

Published online before print November 2, 2006, doi:10.1212/01.wnl.0000249142.82285.d6)
This Article
Right arrow Figures Only
Right arrow Full Text
Right arrow Full Text (PDF)
Right arrow All Versions of this Article:
01.wnl.0000249142.82285.d6v1
68/1/51    most recent
Right arrow Correspondence:
Submit a response
Right arrow Alert me when this article is cited
Right arrow Alert me when Correspondence are posted
Right arrow Alert me if a correction is posted
Right arrow Citation Map
Services
Right arrow Email this article to a friend
Right arrow Similar articles in this journal
Right arrow Similar articles in PubMed
Right arrow Alert me to new issues of the journal
Right arrow Download to citation manager
Right arrow reprints & permissions
Citing Articles
Right arrow Citing Articles via HighWire
Right arrow Citing Articles via Google Scholar
Google Scholar
Right arrow Articles by Mercuri, E.
Right arrow Articles by Brahe, C.
Right arrow Search for Related Content
PubMed
Right arrow PubMed Citation
Right arrow Articles by Mercuri, E.
Right arrow Articles by Brahe, C.
Related Collections
Right arrow Anterior nerve cell disease
Right arrow All Clinical trials
Right arrow Clinical trials Randomized controlled (CONSORT agreement)
NEUROLOGY 2007;68:51-55
© 2007 American Academy of Neurology

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

E. Mercuri, MD, E. Bertini, MD, S. Messina, MD, A. Solari, MD, A. D'Amico, MD, C. Angelozzi, MD, R. Battini, MD, A. Berardinelli, MD, P. Boffi, MD, C. Bruno, MD, C. Cini, MD, F. Colitto, MD, M. Kinali, MD, C. Minetti, MD, T. Mongini, MD, L. Morandi, MD, G. Neri, MD, S. Orcesi, MD, M. Pane, MD, M. Pelliccioni, MD, A. Pini, MD, F. D. Tiziano, MD, M. Villanova, MD, G. Vita, MD and C. Brahe, MD

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.).

Address correspondence and reprint requests to Dr. E. Mercuri, Department of Paediatric Neurology, Catholic University, Largo Gemelli, 00168 Rome, Italy; e-mail: e.mercuri{at}imperial.ac.uk

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.

Editorial, see page 11

This article was previously published in electronic format as an Expedited E-Pub on November 2, 2006, at www.neurology.org.

*Both authors contributed equally.

Disclosure: The authors report no conflicts of interest.

Received February 22, 2006. Accepted in final form August 28, 2006.




This article has been cited by other articles:


Home page
 J Child NeurolHome page
J. Montes, A. M. Gordon, S. Pandya, D. C. De Vivo, and P. Kaufmann
Clinical Outcome Measures in Spinal Muscular Atrophy
J Child Neurol, August 1, 2009; 24(8): 968 - 978.
[Abstract] [PDF]

Home page
 J. Med. Genet.Home page
C Angelozzi, F Borgo, F D Tiziano, A Martella, G Neri, and C Brahe
Salbutamol increases SMN mRNA and protein levels in spinal muscular atrophy cells
J. Med. Genet., January 1, 2008; 45(1): 29 - 31.
[Abstract] [Full Text] [PDF]

Home page
 J Child NeurolHome page
K. J. Swoboda, J. T. Kissel, T. O. Crawford, M. B. Bromberg, G. Acsadi, G. D'Anjou, K. J. Krosschell, S. P. Reyna, M. K. Schroth, C. B. Scott, et al.
Perspectives on Clinical Trials in Spinal Muscular Atrophy
J Child Neurol, August 1, 2007; 22(8): 957 - 966.
[Abstract] [PDF]

Home page
 J Child NeurolHome page
C. J. Sumner
Molecular Mechanisms of Spinal Muscular Atrophy
J Child Neurol, August 1, 2007; 22(8): 979 - 989.
[Abstract] [PDF]

Home page
 NeurologyHome page
P. Kaufmann and R. Finkel
Learning to walk: Challenges for spinal muscular atrophy clinical trials
Neurology, January 2, 2007; 68(1): 11 - 12.
[Full Text] [PDF]


HOME HELP FEEDBACK SUBSCRIPTIONS ARCHIVE SEARCH TABLE OF CONTENTS
Copyright © 2007 by AAN Enterprises, Inc.