Spinal muscular atrophy — the dawning of a new era-Reference-Cited by-同舟云学术

Spinal muscular atrophy — the dawning of a new era

Published:2020-09-25 Issue:11 Volume:16 Page:593-594
ISSN:1759-4758
Container-title:Nature Reviews Neurology
language:en
Short-container-title:Nat Rev Neurol

Author:

Farrar Michelle A.^ORCID,Kiernan Matthew C.^ORCID

Publisher

Springer Science and Business Media LLC

Subject

Cellular and Molecular Neuroscience,Clinical Neurology

Link

https://www.nature.com/articles/s41582-020-00410-7.pdf

Reference10 articles.

1. Coovert, D. D. et al. The survival motor neuron protein in spinal muscular atrophy. Hum. Mol. Genet. 6, 1205–1214 (1997).

2. Finkel, R. S. et al. Nusinersen versus sham control in infantile-onset spinal muscular atrophy. N. Engl. J. Med. 377, 1723–1732 (2017).

3. Canadian Agency for Drugs and Technologies in Health. Nusinersen. CADTH, https://www.cadth.ca/nusinersen (2017).

4. World Health Organization. Improving the transparency of markets for medicines, vaccines, and other health products. WHO, https://apps.who.int/gb/ebwha/pdf_files/WHA72/A72_ACONF2Rev1-en.pdf (2019).

5. Powell, T. & O’Donnell, M. NICE appraisals of rare disease. House of Commons Library, https://researchbriefings.files.parliament.uk/documents/CDP-2019-0022/CDP-2019-0022.pdf (2019).

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