AVXS-101 Gene Replacement Therapy for Spinal Muscular Atrophy: A Comparative Study with a Prospective Natural History Cohort
Samiah Al-Zaidy,Stephen J. Kolb,Linda Lowes,Lindsay N. Alfano,Richard Shell,Kathleen R. Church,Sukumar Nagendran,Douglas M. Sproule,Douglas E. Feltner,Courtney Wells, Francis Orginc,Melissa Menier,James L'Italien,W. David Arnold,John T. Kissel,Brian K. Kaspar,Jerry R. Mendell MOLECULAR THERAPY（2019）
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