Assuring long-term safety of highly effective gene-modulating therapeutics for rare diseases
Author:
Publisher
American Society for Clinical Investigation
Subject
General Medicine
Link
https://www.jci.org/articles/view/152817/files/pdf
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2. Single-dose gene-replacement therapy for spinal muscular atrophy;Mendell;N Engl J Med,2017
3. Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN;Foust;Nat Biotechnol,2010
4. Adeno-associated viral vector-mediated immune responses: understanding barriers to gene delivery;Nidetz;Pharmacol Ther,2020
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3. Experience and Perspectives in the US on the Evolving Treatment Landscape in Spinal Muscular Atrophy;International Journal of General Medicine;2022-09
4. Early treatment is a lifeline for infants with SMA;Nature Medicine;2022-07
5. Onasemnogene abeparvovec for the treatment of spinal muscular atrophy;Expert Opinion on Biological Therapy;2022-05-02
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