Nucleic Acid Therapeutics for Neurological Diseases-Reference-Cited by-同舟云学术

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Nucleic Acid Therapeutics for Neurological Diseases

Published:2019-04 Issue:2 Volume:16 Page:245-247
ISSN:1933-7213
Container-title:Neurotherapeutics
language:en
Short-container-title:Neurotherapeutics

Author:

Watts Jonathan K.^ORCID,Brown Robert H.,Khvorova Anastasia

Funder

National Institute of Neurological Disorders and Stroke

Ono Medical Research Foundation

Publisher

Springer Science and Business Media LLC

Subject

Pharmacology (medical),Clinical Neurology,Pharmacology

Link

http://link.springer.com/content/pdf/10.1007/s13311-019-00736-1.pdf

Reference17 articles.

1. Raal, F.J., Santos, R.D., Blom, D.J. et al. (2010). Mipomersen, an apolipoprotein B synthesis inhibitor, for lowering of LDL cholesterol concentrations in patients with homozygous familial hypercholesterolaemia: a randomised, double-blind, placebo-controlled trial. Lancet, 375, 998–1006.

2. Benson, M.D., Waddington-Cruz, M., Berk, J.L. et al. (2018). Inotersen treatment for patients with hereditary transthyretin amyloidosis. N Engl J Med, 379, 22–31.

3. Adams, D., Gonzalez-Duarte, A., O’Riordan, W.D. et al. (2018). Patisiran, an RNAi therapeutic, for hereditary transthyretin amyloidosis. N Engl J Med, 379, 11–21.

4. Chiriboga, C.A., Swoboda, K.J., Darras, B.T. et al. (2016). Results from a phase 1 study of nusinersen (ISIS-SMN(Rx)) in children with spinal muscular atrophy. Neurology, 86, 890–897.

5. Aartsma-Rus, A. (2017). FDA Approval of nusinersen for spinal muscular atrophy makes 2016 the year of splice modulating oligonucleotides. Nucleic Acid Ther, 27, 67–69.

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