Observational study of spinal muscular atrophy type 2 and 3: functional outcomes over 1 year.

  title={Observational study of spinal muscular atrophy type 2 and 3: functional outcomes over 1 year.},
  author={Petra Kaufmann and Michael P Mcdermott and Basil T. Darras and Richard Finkel and Prabhjot Kaur Kang and Maryam Oskoui and Andrei Constantinescu and Douglas Michael Sproule and A. Reghan Foley and Michele Yang and Rabi Tawil and Wendy Chung and Bill Martens and Jacqueline Montes and Jessica M O'Hagen and Sally L Dunaway and Jean M Flickinger and Janet Quigley and Stuart I. Riley and Allan M. Glanzman and Maryjane Benton and Patricia A. Ryan and Catriona Irvine and Christine L Annis and Hailly E. Butler and Jayson Caracciolo and Megan J. Montgomery and Jonathan D. Marra and Benjamin Koo and Darryl C. De Vivo},
  journal={Archives of neurology},
  volume={68 6},
OBJECTIVE To characterize the short-term course of spinal muscular atrophy (SMA) in a genetically and clinically well-defined cohort of patients with SMA. DESIGN A comprehensive multicenter, longitudinal, observational study. SETTING The Pediatric Neuromuscular Clinical Research Network for SMA, a consortium of clinical investigators at 3 clinical sites. PARTICIPANTS Sixty-five participants with SMA types 2 and 3, aged 20 months to 45 years, were prospectively evaluated. INTERVENTION We… CONTINUE READING

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