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

Authors

John Day

Published

March 18, 2021

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The Lancet Neurology

Topics

DOI

10.1016/s1474-4422(21)00001-6

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

Authors

John Day

Published

March 18, 2021

Share your thoughts on this paper...

Supporters

Support the authors with ResearchCoin

Journal

The Lancet Neurology

Topics

DOI

10.1016/s1474-4422(21)00001-6

Paper

Paper

Paper

Paper

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

Supporters

Journal

Topics

DOI

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

Supporters

Journal

Topics

DOI