Evaluation of SMN Protein, Transcript, and Copy Number in the Biomarkers for Spinal Muscular Atrophy (BforSMA) Clinical Study

• Thomas O. Crawford ,

  * E-mail: tcrawfo@jhmi.edu

  Affiliation Departments of Neurology and Pediatrics, The Johns Hopkins University, Baltimore, Maryland, United States of America

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• Sergey V. Paushkin,

  Affiliation Spinal Muscular Atrophy Foundation, New York, New York, United States of America

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• Dione T. Kobayashi,

  Affiliation Spinal Muscular Atrophy Foundation, New York, New York, United States of America

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• Suzanne J. Forrest,

  Affiliation Spinal Muscular Atrophy Foundation, New York, New York, United States of America

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• Cynthia L. Joyce,

  Affiliation Spinal Muscular Atrophy Foundation, New York, New York, United States of America

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• Richard S. Finkel,

  Affiliation Departments of Neurology and Pediatrics, Children’s Hospital of Philadelphia and University of Pennsylvania School of Medicine, Philadelphia, Pennsylvania, United States of America

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• Petra Kaufmann,

  Affiliation Department of Neurology, Columbia University, New York, New York, United States of America

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• Kathryn J. Swoboda,

  Affiliation Departments of Neurology and Pediatrics, University of Utah School of Medicine, Salt Lake City, Utah, United States of America

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• Danilo Tiziano,

  Affiliation Instituto di Genetica Medica, Università Cattolica S. Cuore, Roma, Italy

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• Rosa Lomastro,

  Affiliation Instituto di Genetica Medica, Università Cattolica S. Cuore, Roma, Italy

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• Rebecca H. Li,

  Affiliation New England Research Institutes, Inc., Watertown, Massachusetts, United States of America

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• Felicia L. Trachtenberg,

  Affiliation New England Research Institutes, Inc., Watertown, Massachusetts, United States of America

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• Thomas Plasterer,

  Affiliation Department of Chemistry and Chemical Biology, Northeastern University, Boston, Massachusetts, United States of America

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• Karen S. Chen,

  Affiliation Spinal Muscular Atrophy Foundation, New York, New York, United States of America

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• on behalf of the Pilot Study of Biomarkers for Spinal Muscular Atrophy (BforSMA) Trial Group

  ¶Members of the BforSMA Trial Group are listed in List S1.

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