O.40Intrathecal administration of onasemnogene abeparvovec gene-replacement therapy (GRT) for spinal muscular atrophy type 2 (SMA2): phase 1/2a study (STRONG)

Neuromuscular Disorders ◽

10.1016/j.nmd.2019.06.594 ◽

2019 ◽

Vol 29 ◽

pp. S207 ◽

Author(s):

R. Finkel ◽

J. Day ◽

B. Darras ◽

N. Kuntz ◽

A. Connolly ◽

...

Keyword(s):

Spinal Muscular Atrophy ◽

Replacement Therapy ◽

Muscular Atrophy ◽

Phase 1 ◽

Gene Replacement ◽

Spinal Muscular Atrophy Type ◽

Gene Replacement Therapy

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

Journal of the Neurological Sciences ◽

10.1016/j.jns.2019.10.322 ◽

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

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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

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AVXS-101 Phase 1 Gene-Replacement Therapy (GRT) Clinical Trial in Spinal Muscular Atrophy Type 1 (SMA1): 24-Month Event-Free Survival and Achievement of Developmental Milestones

10.1055/s-0039-1685425 ◽

2019 ◽

Author(s):

J.R. Mendell ◽

S. Al-Zaidy ◽

R. Shell ◽

W.D. Arnold ◽

L. Rodino-Klapac ◽

...

Keyword(s):

Clinical Trial ◽

Spinal Muscular Atrophy ◽

Muscular Atrophy ◽

Phase 1 ◽

Gene Replacement ◽

Event Free Survival ◽

Free Survival ◽

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

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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

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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 ◽

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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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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