AVXS-101 Phase 1 gene therapy clinical trial in SMA Type 1: Interim data demonstrates improvements in supportive care use

2017 ◽  
Vol 21 ◽  
pp. e14 ◽  
Author(s):  
Richard Shell ◽  
Samiah Al-Zaidy ◽  
W. Dave Arnold ◽  
Louise Rodino-Klapac ◽  
Thomas W. Prior ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Gene Therapy ◽  
Supportive Care ◽  
Phase 1 ◽  
Therapy Clinical Trial

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

2017 ◽  
Vol 27 ◽  
pp. S208
Author(s):  
R. Shell ◽  
S. Al-Zaidy ◽  
W. Arnold ◽  
L. Rodino-Klapac ◽  
T. Prior ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Gene Therapy ◽  
Nutritional Support ◽  
Phase 1 ◽  
Therapy Clinical Trial

AVXS-101 Phase 1 gene therapy clinical trial in SMA Type 1: Event free survival and achievement of developmental milestones

2017 ◽  
Vol 21 ◽  
pp. e13-e14 ◽  
Author(s):  
Jerry R. Mendell ◽  
Samiah Al-Zaidy ◽  
Richard Shell ◽  
W. David Arnold ◽  
Louise Rodino-Klapac ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Gene Therapy ◽  
Phase 1 ◽  
Developmental Milestones ◽  
Event Free Survival ◽  
Free Survival ◽  
Therapy Clinical Trial

scholarly journals Evaluation of Leberʼs hereditary optic neuropathy patients prior to a gene therapy clinical trial

Medicine ◽  
2016 ◽  
Vol 95 (40) ◽  
pp. e5110 ◽  
Author(s):  
Shuo Yang ◽  
Hong Yang ◽  
Si-qi Ma ◽  
Shuai-shuai Wang ◽  
Heng He ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Gene Therapy ◽  
Optic Neuropathy ◽  
Therapy Clinical Trial ◽  
Hereditary Optic Neuropathy
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