AVXS-101 phase 1 gene therapy clinical trial in SMA Type 1: decreased need of ventilatory and nutritional support at End-of-Study

Authors :: R. Shell
S. Al-Zaidy
W. Arnold
L. Rodino-Klapac
T. Prior
L. Lowes
L. Alfano
K. Berry
K. Church
J. Kissel
S. Nagendran
J. L'Italien
D. Sproule
C. Wells
A. Burghes
K. Foust
K. Meyer
S. Likhite
B. Kaspar
J. Mendell
Source :: Neuromuscular Disorders. 27:S208
Publication Year :: 2017
Publisher :: Elsevier BV, 2017.

Subjects :: Clinical trial
medicine.medical_specialty
Neurology
business.industry
Genetic enhancement
Pediatrics, Perinatology and Child Health
Physical therapy
medicine
Neurology (clinical)
business
SMA
Genetics (clinical)

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