Spinal Muscular Atrophy
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Spinal Muscular Atrophy A Timely Review
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Spinal Muscular Atrophy
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Nusinersen in Adult Patients with Spinal Muscular Atrophy
Neurol 95:e413-e416, Moshe-Lilie, O.,et al, 2020
Nusinersen Versus Sham Control in Later-Onset Spinal Muscular Atrophy
NEJM 378:625-635, Mercuri, E.,et al, 2018
Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy
NEJM 377:1713-1722,1786, Mendell, J.R.,et al, 2017
Nusinersen Versus Sham Control in Infantile-Onset Spinal Muscular Atrophy
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The Floppy Infant: Evaluation of Hypotonia
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Molecular Basis of the Neurodegenerative Disorders
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From Enigmatic to Problematic:The New Molecular Genetics of Childhood Spinal Muscular Atrophy
Neurol 46:335-340, Crawford,T.O., 1996
Clinicopath Conf
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Ataxia-Telangiectasia:An Interdisciplinary Approach to Pathogenesis
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