Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy

Jerry R. Mendell; Samiah Al-Zaidy; Richard Shell; W. Dave Arnold; Louise R. Rodino-Klapac; Thomas W. Prior; Linda Lowes; Lindsay Alfano; Katherine Berry; Kathleen Church; John T. Kissel; Sukumar Nagendran; James L’Italien; Douglas M. Sproule; Courtney Wells; Jessica A. Cardenas; Marjet D. Heitzer; Allan Kaspar; Sarah Corcoran; Lyndsey Braun; Shibi Likhite; Carlos Miranda; Kathrin Meyer; K.D. Foust; Arthur H.M. Burghes; Brian K. Kaspar

doi:10.1056/nejmoa1706198

Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy

New England Journal of Medicine ◽

10.1056/nejmoa1706198 ◽

2017 ◽

Vol 377 (18) ◽

pp. 1713-1722 ◽

Cited By ~ 668

Author(s):

Jerry R. Mendell ◽

Samiah Al-Zaidy ◽

Richard Shell ◽

W. Dave Arnold ◽

Louise R. Rodino-Klapac ◽

...

Keyword(s):

Single Dose ◽

Spinal Muscular Atrophy ◽

Replacement Therapy ◽

Muscular Atrophy ◽

Gene Replacement ◽

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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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 ◽

Cited By ~ 1

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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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 ◽

Cited By ~ 1

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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European ad-hoc consensus statement on gene replacement therapy for spinal muscular atrophy

European Journal of Paediatric Neurology ◽

10.1016/j.ejpn.2020.07.001 ◽

2020 ◽

Vol 28 ◽

pp. 38-43 ◽

Cited By ~ 4

Author(s):

Janbernd Kirschner ◽

Nina Butoianu ◽

Nathalie Goemans ◽

Jana Haberlova ◽

Anna Kostera-Pruszczyk ◽

...

Keyword(s):

Spinal Muscular Atrophy ◽

Replacement Therapy ◽

Ad Hoc ◽

Consensus Statement ◽

Muscular Atrophy ◽

Gene Replacement ◽

Gene Replacement Therapy

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P.350Onasemnogene abeparvovec gene-replacement therapy (GRT) in pre-symptomatic spinal muscular atrophy (SMA)

Neuromuscular Disorders ◽

10.1016/j.nmd.2019.06.512 ◽

2019 ◽

Vol 29 ◽

pp. S183

Author(s):

M. Schultz ◽

K. Swoboda ◽

M. Farrar ◽

H. McMillan ◽

J. Parsons ◽

...

Keyword(s):

Spinal Muscular Atrophy ◽

Replacement Therapy ◽

Muscular Atrophy ◽

Gene Replacement ◽

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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Thrombotic Microangiopathy (TMA) after Gene Replacement Therapy (GRT) Due to Spinal Muscular Atrophy: Case Summary and Recommendations for Treatment

10.1055/s-0041-1739642 ◽

2021 ◽

Author(s):

D. Witte ◽

H. Hartmann ◽

J. Drube ◽

D. Haffner ◽

S. Illsinger

Keyword(s):

Spinal Muscular Atrophy ◽

Replacement Therapy ◽

Thrombotic Microangiopathy ◽

Muscular Atrophy ◽

Gene Replacement ◽

Case Summary ◽

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

Download Full-text