scholarly journals Intrathecal administration of AVXS-101 gene-replacement therapy (GRT) for spinal muscular atrophy type 2 (SMA2): Phase 1/2A study (strong)

2019 ◽  
Vol 405 ◽  
pp. 57
Author(s):  
R.S. Finkel ◽  
J.W. Day ◽  
B.T. Darras ◽  
N.L. Kuntz ◽  
A.M. Connolly ◽  
...  
Keyword(s):  
Spinal Muscular Atrophy ◽  
Replacement Therapy ◽  
Muscular Atrophy ◽  
Phase 1 ◽  
Gene Replacement ◽  
Intrathecal Administration ◽  
Spinal Muscular Atrophy Type ◽  
Gene Replacement Therapy

scholarly journals Health outcomes in spinal muscular atrophy type 1 following AVXS‐101 gene replacement therapy

2018 ◽  
Vol 54 (2) ◽  
pp. 179-185 ◽  
Author(s):  
Samiah Al‐Zaidy ◽  
A. Simon Pickard ◽  
Kavitha Kotha ◽  
Lindsay N. Alfano ◽  
Linda Lowes ◽  
...  
Keyword(s):  
Health Outcomes ◽  
Spinal Muscular Atrophy ◽  
Replacement Therapy ◽  
Muscular Atrophy ◽  
Gene Replacement ◽  
Spinal Muscular Atrophy Type ◽  
Gene Replacement Therapy
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