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

Samiah Al‐Zaidy; A. Simon Pickard; Kavitha Kotha; Lindsay N. Alfano; Linda Lowes; Grace Paul; Kathleen Church; Kelly Lehman; Douglas M. Sproule; Omar Dabbous; Benit Maru; Katherine Berry; W. David Arnold; John T. Kissel; Jerry R. Mendell; Richard Shell

doi:10.1002/ppul.24203

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 ◽

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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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

10.1055/s-0039-1698171 ◽

2019 ◽

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

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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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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 ◽

Replacement Therapy ◽

Muscular Atrophy ◽

Gene Replacement ◽

Spinal Muscular Atrophy Type ◽

Gene Replacement Therapy

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Single-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies

Case Medical Research ◽

10.31525/ct1-nct03837184 ◽

2000 ◽

Author(s):

Keyword(s):

Single Dose ◽

Spinal Muscular Atrophy ◽

Replacement Therapy ◽

Muscular Atrophy ◽

Gene Replacement ◽

Spinal Muscular Atrophy Type ◽

Gene Replacement Therapy

Download Full-text

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

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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 ◽

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

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 ◽

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

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