Abstract:
INTRODUCTION: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.
journal_name
Muscle Nervejournal_title
Muscle & nerveauthors
Kissel JT,Elsheikh B,King WM,Freimer M,Scott CB,Kolb SJ,Reyna SP,Crawford TO,Simard LR,Krosschell KJ,Acsadi G,Schroth MK,D'Anjou G,LaSalle B,Prior TW,Sorenson S,Maczulski JA,Swoboda KJ,Project Cure Spinal Muscular Atrdoi
10.1002/mus.23904subject
Has Abstractpub_date
2014-02-01 00:00:00pages
187-92issue
2eissn
0148-639Xissn
1097-4598journal_volume
49pub_type
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