Details

Autor(en) / Beteiligte

• 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.

Titel

SMA valiant trial: A prospective, double-blind, placebo-controlled trial of valproic acid in ambulatory adults with spinal muscular atrophy

Ist Teil von

• Muscle & nerve, 2014-02, Vol.49 (2), p.187-192

Ort / Verlag

United States: Blackwell Publishing Ltd

Erscheinungsjahr

2014

Link zum Volltext

Quelle

MEDLINE

Beschreibungen/Notizen

• 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. Muscle Nerve 49: 187–192, 2014