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SPINAL MUSCULAR ATROPHY DISEASE MECHANISMS AND THERAPY Edited by Charlotte J. Sumner Johns Hopkins University School of Medicine, Baltimore, MD, United States Sergey PauShkin Spinal Muscular Atrophy (SMA) Foundation, New York, NY, United States Chien-Ping ko University of Southern California, Los Angeles, CA, United States AMSTERDAM • BOSTON • HEIDELBERG • LONDON NEW YORK • OXFORD • PARIS • SAN DIEGO SAN FRANCISCO • SINGAPORE • SYDNEY • TOKYO Academic Press is an imprint of Elsevier SPINAL MUSCULAR ATROPHY: DISEASE MECHANISMS AND THERAPY