Clinical Trial and Postmarketing Safety of Onasemnogene Abeparvovec Therapy-Reference-Cited by-同舟云学术

Clinical Trial and Postmarketing Safety of Onasemnogene Abeparvovec Therapy

Published:2021-08-12 Issue:10 Volume:44 Page:1109-1119
ISSN:0114-5916
Container-title:Drug Safety
language:en
Short-container-title:Drug Saf

Author:

Day John W.,Mendell Jerry R.,Mercuri Eugenio,Finkel Richard S.,Strauss Kevin A.,Kleyn Aaron,Tauscher-Wisniewski Sitra,Tukov Francis Fonyuy,Reyna Sandra P.,Chand Deepa H.

Funder

novartis gene therapies, inc.

Publisher

Springer Science and Business Media LLC

Subject

Pharmacology (medical),Pharmacology,Toxicology

Link

https://link.springer.com/content/pdf/10.1007/s40264-021-01107-6.pdf

Reference34 articles.

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2. Kolb S, Kissel JT. Spinal muscular atrophy. Neurol Clin. 2015;33:831–46.

3. Darras BT, Monani UR, De Vivo DC, et al. Genetic disorders affecting the motor neuron: spinal muscular atrophy. In: Swaiman KF, et al., editors. Swaiman’s pediatric neurology: principles and practice. 6th ed. Edinburgh: Elsevier; 2017. p. 1057–64.

4. Mendell JR, Al-Zaidy S, Shell R, et al. Single-dose gene-replacement therapy for spinal muscular atrophy. N Engl J Med. 2017;377:1713–22.

5. Day JW, Finkel RS, Chiriboga CA, et al. Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy with two copies of SMN2 (STR1VE): an open-label, single-arm, phase 3 study. Lancet Neurol. 2021;20:284–93.

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