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Study Design of STR1VE-EU, a Phase 3 Trial of AVXS-101 Gene-Replacement Therapy (GRT) in Patients With Spinal Muscular Atrophy Type 1 (SMA1) in Europe
Mapping Intimacies
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10.1055/s-0039-1698171
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2019
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Author(s):
Francesco Muntoni
◽
Giovanni Baranello
◽
Claudio Bruno
◽
Stefania Corti
◽
Riccardo Masson
◽
...
Keyword(s):
Spinal Muscular Atrophy
◽
Replacement Therapy
◽
Study Design
◽
Muscular Atrophy
◽
Gene Replacement
◽
Phase 3
◽
Spinal Muscular Atrophy Type
◽
Gene Replacement Therapy
Download Full-text
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References
P.349Onasemnogene abeparvovec gene-replacement therapy (GRT) for spinal muscular atrophy Type 1 (SMA1): Pivotal phase 3 study (STR1VE) update
Neuromuscular Disorders
◽
10.1016/j.nmd.2019.06.511
◽
2019
◽
Vol 29
◽
pp. S183
Author(s):
J. Day
◽
C. Chiriboga
◽
T. Crawford
◽
B. Darras
◽
R. Finkel
◽
...
Keyword(s):
Spinal Muscular Atrophy
◽
Replacement Therapy
◽
Muscular Atrophy
◽
Gene Replacement
◽
Phase 3
◽
Pivotal Phase
◽
Spinal Muscular Atrophy Type
◽
Gene Replacement Therapy
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Onasemnogene Abeparvovec Gene-Replacement Therapy (GRT) for Spinal Muscular Atrophy Type 1 (SMA1): Pivotal Phase 3 Study (STR1VE) Update
10.1055/s-0039-1698170
◽
2019
◽
Author(s):
John W. Day
◽
Claudia A. Chiriboga
◽
Thomas O. Crawford
◽
Basil T. Darras
◽
Richard S. Finkel
◽
...
Keyword(s):
Spinal Muscular Atrophy
◽
Replacement Therapy
◽
Muscular Atrophy
◽
Gene Replacement
◽
Phase 3
◽
Pivotal Phase
◽
Spinal Muscular Atrophy Type
◽
Gene Replacement Therapy
Download Full-text
AVXS-101 Gene Replacement Therapy (GRT) for Spinal Muscular Atrophy Type 1 (SMA1): Pivotal Phase 3 Study (STR1VE) Update
10.1164/ajrccm-conference.2019.199.1_meetingabstracts.a2789
◽
2019
◽
Author(s):
R. Shell
◽
J.W. Day
◽
C. Chiriboga
◽
T.O. Crawford
◽
B.T. Darras
◽
...
Keyword(s):
Spinal Muscular Atrophy
◽
Replacement Therapy
◽
Muscular Atrophy
◽
Gene Replacement
◽
Phase 3
◽
Pivotal Phase
◽
Spinal Muscular Atrophy Type
◽
Gene Replacement Therapy
Download Full-text
Onasemnogene abeparvovec gene-replacement therapy (GRT) for spinal muscular atrophy type 1 (SMA1): Global pivotal phase 3 study program (STR1VE-US, STR1VE-EU, STR1VE-AP)
Journal of the Neurological Sciences
◽
10.1016/j.jns.2019.10.1338
◽
2019
◽
Vol 405
◽
pp. 277-278
Author(s):
E. Mercuri
◽
G. Baranello
◽
J.W. Day
◽
C. Bruno
◽
S. Corti
◽
...
Keyword(s):
Spinal Muscular Atrophy
◽
Replacement Therapy
◽
Muscular Atrophy
◽
Gene Replacement
◽
Phase 3
◽
Pivotal Phase
◽
Study Program
◽
Spinal Muscular Atrophy Type
◽
Gene Replacement Therapy
Download Full-text
S61 Onasemnogene abeparvovec gene-replacement therapy (GRT) for spinal muscular atrophy type 1 (SMA1): preliminary pulmonary and ventilatory findings from the phase 3 study (STR1VE)
10.1136/thorax-2019-btsabstracts2019.67
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2019
◽
Author(s):
R Shell
◽
JW Day
◽
CA Chiriboga
◽
TO Crawford
◽
BT Darras
◽
...
Keyword(s):
Spinal Muscular Atrophy
◽
Replacement Therapy
◽
Muscular Atrophy
◽
Gene Replacement
◽
Phase 3
◽
Spinal Muscular Atrophy Type
◽
Gene Replacement Therapy
Download Full-text
Onasemnogene Abeparvovec-xioi Gene-Replacement Therapy for Spinal Muscular Atrophy Type 1: Pulmonary and Ventilatory Findings from the Pivotal Phase 3 US Study (STR1VE)
10.1164/ajrccm-conference.2020.201.1_meetingabstracts.a5974
◽
2020
◽
Author(s):
R. Shell
◽
J.W. Day
◽
C.A. Chiriboga
◽
T.O. Crawford
◽
B.T. Darras
◽
...
Keyword(s):
Spinal Muscular Atrophy
◽
Replacement Therapy
◽
Muscular Atrophy
◽
Gene Replacement
◽
Phase 3
◽
Pivotal Phase
◽
Spinal Muscular Atrophy Type
◽
Gene Replacement Therapy
Download Full-text
The value of onasemnogene abeparvovec (AVXS-101) gene-replacement therapy for spinal muscular atrophy type 1
Journal of the Neurological Sciences
◽
10.1016/j.jns.2019.10.327
◽
2019
◽
Vol 405
◽
pp. 59
Author(s):
O. Dabbous
◽
D.M. Sproule
◽
D.E. Feltner
◽
M. Droege
◽
F. Khan
◽
...
Keyword(s):
Spinal Muscular Atrophy
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Replacement Therapy
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Muscular Atrophy
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Gene Replacement
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Spinal Muscular Atrophy Type
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Gene Replacement Therapy
Download Full-text
Single-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies
Case Medical Research
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10.31525/ct1-nct03837184
◽
2000
◽
Author(s):
Keyword(s):
Single Dose
◽
Spinal Muscular Atrophy
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Replacement Therapy
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Muscular Atrophy
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Gene Replacement
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Spinal Muscular Atrophy Type
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Gene Replacement Therapy
Download Full-text
Health outcomes in spinal muscular atrophy type 1 following AVXS‐101 gene replacement therapy
Pediatric Pulmonology
◽
10.1002/ppul.24203
◽
2018
◽
Vol 54
(2)
◽
pp. 179-185
◽
Cited By ~ 36
Author(s):
Samiah Al‐Zaidy
◽
A. Simon Pickard
◽
Kavitha Kotha
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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
Download Full-text
PRO2 THE VALUE OF ONASEMNOGENE ABEPARVOVEC GENE-REPLACEMENT THERAPY IN IMPROVING SURVIVAL AND MOTOR FUNCTION AND DECREASING VENTILATORY SUPPORT AND HOSPITALIZATION CONTRASTED TO NATURAL HISTORY FOR SPINAL MUSCULAR ATROPHY TYPE 1
Value in Health
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10.1016/j.jval.2019.09.2334
◽
2019
◽
Vol 22
◽
pp. S841
Author(s):
O. Dabbous
◽
D.M. Sproule
◽
D.E. Feltner
◽
F.G. Ogrinc
◽
M. Menier
◽
...
Keyword(s):
Natural History
◽
Spinal Muscular Atrophy
◽
Replacement Therapy
◽
Motor Function
◽
Muscular Atrophy
◽
Ventilatory Support
◽
Gene Replacement
◽
Spinal Muscular Atrophy Type
◽
Gene Replacement Therapy
Download Full-text
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