Original articleProspective study of spinal muscular atrophy before age 6 years☆
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2019, Neuromuscular DisordersCitation Excerpt :The overall treatment effect in terms of change from baseline on the 3 scales across all visits was significantly better in the youngest patients (<6 years). These data must be considered with caution since children with SMA without treatment might experience improvements in motor function during their first years of life [43,47] and a possible placebo effect cannot be ruled out, although the improvement observed is different from the typical temporary early response associated with a placebo effect. The main limitation of the study was the absence of a control group.
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This work was funded by the Muscular Dystrophy Association of America, Inc, and was presented in part at the 1991 meeting of the Child Neurology Society in Portland, Oregon.
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The DCN/SMA Study Group includes: Barry S. Russman, MD, University of Connecticut and Newington Children's Hospital, Newington, CT; Laurie Zimmerman, MA, OTR, and Betsy Perkins, RPT, Newington Children's Hospital, Newington, CT; Frederick J. Samaha, MD and Jeanne Bender-Dragoo, RN, University of Cincinnati Medical Center, Cincinnati, OH; C. Ralph Buncher, ScD, and Michael White, MS, University of Cincinnati Medical Center, Cincinnati, OH; Lisa Barker, LPT, and Karen Burhans, MOT, OTRL, Children's Hospital Medical Center, Cincinnati, OH; Susan T. Iannaccone, MD, University of Texas Southwestern Medical Center, Dallas, TX; Cindy Smith, BS, Richard H. Browne, PhD, and David Ross, MA, Texas Scottish Rite Hospital for Children, Dallas, TX.