Current Treatment Options in Neurology—SMA Therapeutics

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作者
Megan A. Waldrop
Stephen J. Kolb
机构
[1] Nationwide Children’s Hospital,Center for Gene Therapy
[2] The Ohio State University Wexner Medical Center,Department of Pediatrics
[3] The Ohio State University Wexner Medical Center,Department of Neurology
[4] The Ohio State University Wexner Medical Center,Department of Biological Chemistry & Pharmacology
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关键词
Spinal muscular atrophy; Antisense oligonucleotide; Nusinersen; Gene therapy;
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