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Pediatr Neurol. 2020 Jan;102:1-2. doi: 10.1016/j.pediatrneurol.2019.07.018. Epub 2019 Aug 6.

The End of the Beginning: The Journey to Molecular Therapies for Spinal Muscular Atrophy.

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1
Department of Pediatrics, University of Florida College of Medicine, Gainesville, Florida.
2
Division of Pediatric Neurology, Department of Pediatrics, University of Florida College of Medicine, Gainesville, Florida; Department of Neurology, University of Florida College of Medicine, Gainesville, Florida; Genetics Institute and Myology Institute, University of Florida, Gainesville, Florida. Electronic address: pbkang@ufl.edu.

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