Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy type 1 (STR1VE-EU): an open-label, single-arm, multicentre, phase 3 trial

Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy in patients with two copies of SMN2 (STR1VE): an open-label, single-arm, multicentre, phase 3 trial

The Lancet Neurology ◽

10.1016/s1474-4422(21)00001-6 ◽

2021 ◽

Vol 20 (4) ◽

pp. 284-293

Author(s):

John W Day ◽

Richard S Finkel ◽

Claudia A Chiriboga ◽

Anne M Connolly ◽

Thomas O Crawford ◽

...

Keyword(s):

Gene Therapy ◽

Spinal Muscular Atrophy ◽

Muscular Atrophy ◽

Open Label ◽

Phase 3 ◽

Multicentre Phase

S12 Onasemnogene abeparvovec gene therapy for spinal muscular atrophy type 1: phase 3 study (STR1VE-US)

10.1136/thorax-2020-btsabstracts.18 ◽

2021 ◽

Author(s):

RS Finkel ◽

AM Connolly ◽

BT Darras ◽

ST Iannaccone ◽

NL Kuntz ◽

...

Keyword(s):

Gene Therapy ◽

Spinal Muscular Atrophy ◽

Muscular Atrophy ◽

Phase 3 ◽

Spinal Muscular Atrophy Type

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 ◽

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 ◽

S11 Long-term follow-up of the phase 1 START trial of onasemnogene abeparvovec gene therapy in spinal muscular atrophy type 1

10.1136/thorax-2020-btsabstracts.17 ◽

2021 ◽

Cited By ~ 1

Author(s):

JR Mendell ◽

R Shell ◽

KJ Lehman ◽

M McColly ◽

LP Lowes ◽

...

Keyword(s):

Gene Therapy ◽

Spinal Muscular Atrophy ◽

Muscular Atrophy ◽

Phase 1 ◽

Long Term Follow Up ◽

Start Trial

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 ◽

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 ◽

480. Gene Therapy for Spinal Muscular Atrophy Type 1 Shows Potential to Improve Survival and Motor Functional Outcomes

Molecular Therapy ◽

10.1016/s1525-0016(16)33289-0 ◽

2016 ◽

Vol 24 ◽

pp. S190 ◽

Cited By ~ 14

Author(s):

Jerry R. Mendell ◽

Samiah Al-Zaidy ◽

Richard Shell ◽

W Dave Arnold ◽

Louise Rodino-Klapac ◽

...

Keyword(s):

Gene Therapy ◽

Spinal Muscular Atrophy ◽

Functional Outcomes ◽

Muscular Atrophy ◽

Spinal Muscular Atrophy Type

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 ◽

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 ◽