Overview of Current Drugs and Molecules in Development for Spinal Muscular Atrophy Therapy
Author:
Funder
Wellcome Trust
Publisher
Springer Science and Business Media LLC
Subject
Pharmacology (medical)
Link
http://link.springer.com/article/10.1007/s40265-018-0868-8/fulltext.html
Reference95 articles.
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3. Lefebvre S, Burglen L, Reboullet S, Clermont O, Burlet P, Viollet L, et al. Identification and characterization of a spinal muscular atrophy-determining gene. Cell. 1995;80(1):155–65.
4. Aartsma-Rus A. FDA approval of nusinersen for spinal muscular atrophy makes 2016 the year of splice modulating oligonucleotides. Nucleic Acid Ther. 2017;27(2):67–9.
5. Hoy SM. Nusinersen: first global approval. Drugs. 2017;77(4):473–9.
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