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Onasemnogene Abeparvovec-xioi Gene Therapy in Presymptomatic Spinal Muscular Atrophy (SMA): SPR1NT Study Update
Onasemnogene Abeparvovec-xioi Gene Therapy in Presymptomatic Spinal Muscular Atrophy (SMA): SPR1NT Study Update
2020
Kevin A. Strauss
Michelle A. Farrar
K.J. Swoboda
K. Saito
Claudia A. Chiriboga
Richard S. Finkel
Susan T. Iannaccone
J.M. Krueger
Jennifer M. Kwon
H.J. McMillan
Laurent Servais
Jerry R. Mendell
Julie Parsons
M. Scoto
Perry B. Shieh
C. Zaidman
M. Schultz
F. Baldinetti
F. Ogrinc
S. Kavanagh
Douglas E. Feltner
S. Tauscher-Wisniewski
B.E. McGill
Douglas M. Sproule
Francesco Muntoni
Keywords:
Pathology
Spinal muscular atrophy
Medicine
SMA*
Genetic enhancement
Correction
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