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

Mapping Intimacies ◽

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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Gene-Replacement Therapy (GRT) in Spinal Muscular Atrophy Type 1 (SMA1): Long-Term Follow-Up From the Onasemnogene Abeparvovec Phase 1/2 Clinical Trial

10.1055/s-0039-1698259 ◽

2019 ◽

Author(s):

Jerry R. Mendell ◽

Richard Shell ◽

Kelly J. Lehman ◽

Markus McColly ◽

Linda P. Lowes ◽

...

Keyword(s):

Clinical Trial ◽

Muscular Atrophy ◽

Phase 1 ◽

Gene Replacement ◽

Spinal Muscular Atrophy Type ◽

Gene Replacement Therapy ◽

Long Term Follow Up

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Gene-replacement therapy (GRT) in spinal muscular atrophy type 1 (SMA1): Long-term follow-up from the onasemnogene abeparvovec phase 1/2A clinical trial

Journal of the Neurological Sciences ◽

10.1016/j.jns.2019.10.1322 ◽

2019 ◽

Vol 405 ◽

pp. 271

Author(s):

J.R. Mendell ◽

R. Shell ◽

K.J. Lehman ◽

M. McColly ◽

L.P. Lowes ◽

...

Keyword(s):

Clinical Trial ◽

Muscular Atrophy ◽

Phase 1 ◽

Gene Replacement ◽

Spinal Muscular Atrophy Type ◽

Gene Replacement Therapy ◽

Long Term Follow Up

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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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PRO10 USE OF SINGLE DOSE GENE-REPLACEMENT THERAPY FOR THE TREATMENT OF SPINAL MUSCULAR ATROPHY TYPE 1: A UNITED STATES PAYER BUDGET IMPACT ANALYSIS

Value in Health ◽

10.1016/j.jval.2019.04.1644 ◽

2019 ◽

Vol 22 ◽

pp. S336-S337 ◽

Author(s):

D. Malone ◽

B. Miller ◽

R. Dean ◽

R. Arjunji ◽

I.S. Jensen ◽

...

Keyword(s):

United States ◽

Single Dose ◽

Spinal Muscular Atrophy ◽

Impact Analysis ◽

Budget Impact ◽

Muscular Atrophy ◽

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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P.351Gene-replacement therapy (GRT) in spinal muscular atrophy type 1 (SMA1): long-term follow-up from the onasemnogene abeparvovec phase 1/2a clinical trial

Neuromuscular Disorders ◽

10.1016/j.nmd.2019.06.513 ◽

2019 ◽

Vol 29 ◽

pp. S184

Author(s):

J. Mendell ◽

R. Shell ◽

K. Lehman ◽

M. McColly ◽

L. Lowes ◽

...

Keyword(s):

Clinical Trial ◽

Spinal Muscular Atrophy ◽

Replacement Therapy ◽

Muscular Atrophy ◽

Phase 1 ◽

Spinal Muscular Atrophy Type ◽

Long Term Follow Up

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ND2 COST-UTILITY ANALYSIS OF SINGLE DOSE GENE-REPLACEMENT THERAPY FOR SPINAL MUSCULAR ATROPHY TYPE 1 COMPARED TO CHRONIC NUSINERSEN TREATMENT

Value in Health ◽

10.1016/j.jval.2019.04.060 ◽

2019 ◽

Vol 22 ◽

pp. S42-S43 ◽

Author(s):

D. Malone ◽

R. Dean ◽

B. Miller ◽

R. Arjunji ◽

D.E. Feltner ◽

...

Keyword(s):

Single Dose ◽

Spinal Muscular Atrophy ◽

Muscular Atrophy ◽

Gene Replacement ◽

Cost Utility ◽

Cost Utility Analysis ◽

Utility Analysis ◽

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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