SMA CARNI-VAL TRIAL PART II: A Prospective, Single-Armed Trial of L-Carnitine and Valproic Acid in Ambulatory Children with Spinal Muscular Atrophy

BACKGROUND: Multiple lines of evidence have suggested that valproic acid (VPA) might benefit patients with spinal muscular atrophy (SMA). The SMA CARNIVAL TRIAL was a two part prospective trial to evaluate oral VPA and l-carnitine in SMA children. Part 1 targeted non-ambulatory children ages 2–8 in...

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Auteurs principaux: Kissel, John T., Scott, Charles B., Reyna, Sandra P., Crawford, Thomas O., Simard, Louise R., Krosschell, Kristin J., Acsadi, Gyula, Elsheik, Bakri, Schroth, Mary K., D'Anjou, Guy, LaSalle, Bernard, Prior, Thomas W., Sorenson, Susan, Maczulski, Jo Anne, Bromberg, Mark B., Chan, Gary M., Swoboda, Kathryn J.
Format: Artigo
Langue:Inglês
Publié: Public Library of Science 2011
Sujets:
Research Article
Accès en ligne:https://ncbi.nlm.nih.gov/pmc/articles/PMC3130730/
https://ncbi.nlm.nih.gov/pubmed/21754985
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0021296
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