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SMA valiant trial: A prospective, double‐blind, placebo‐controlled trial of valproic acid in ambulatory adults with spinal muscular atrophy
Author(s) -
Kissel John T.,
Elsheikh Bakri,
King Wendy M.,
Freimer Miriam,
Scott Charles B.,
Kolb Stephen J.,
Reyna Sandra P.,
Crawford Thomas O.,
Simard Louise R.,
Krosschell Kristin J.,
Acsadi Gyula,
Schroth Mary K.,
D'Anjou Guy,
LaSalle Bernard,
Prior Thomas W.,
Sorenson Susan,
Maczulski Jo Anne,
Swoboda Kathryn J.
Publication year - 2014
Publication title -
muscle and nerve
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.025
H-Index - 145
eISSN - 1097-4598
pISSN - 0148-639X
DOI - 10.1002/mus.23904
Subject(s) - spinal muscular atrophy , sma* , medicine , placebo , ambulatory , valproic acid , isometric exercise , physical therapy , clinical trial , anesthesia , epilepsy , pathology , alternative medicine , mathematics , disease , combinatorics , psychiatry
: An open‐label trial suggested that valproic acid (VPA) improved strength in adults with spinal muscular atrophy (SMA). We report a 12‐month, double‐blind, cross‐over study of VPA in ambulatory SMA adults. Methods : There were 33 subjects, aged 20–55 years, included in this investigation. After baseline assessment, subjects were randomized to receive VPA (10–20 mg/kg/day) or placebo. At 6 months, patients were switched to the other group. Assessments were performed at 3, 6, and 12 months. The primary outcome was the 6‐month change in maximum voluntary isometric contraction testing with pulmonary, electrophysiological, and functional secondary outcomes. Results : Thirty subjects completed the study. VPA was well tolerated, and compliance was good. There was no change in primary or secondary outcomes at 6 or 12 months. Conclusions : VPA did not improve strength or function in SMA adults. The outcomes used are feasible and reliable and can be employed in future trials in SMA adults. Muscle Nerve 49 : 187–192, 2014