RNA-based drug discovery for spinal muscular atrophy: a story of small molecules and antisense oligonucleotides
Author:
Affiliation:
1. Charles River Laboratories, Leiden, the Netherlands
2. Charles River Laboratories, Saffron Walden, UK
3. Charles River Laboratories, Reno, Nevada, USA
Publisher
Informa UK Limited
Subject
Drug Discovery
Link
https://www.tandfonline.com/doi/pdf/10.1080/17460441.2022.2149733
Reference56 articles.
1. CLASSIFICATION OF SPINAL MUSCULAR ATROPHIES
2. A multi-source approach to determine SMA incidence and research ready population
3. Prior TW, Leach ME, Finanger E, et al. Spinal Muscular Atrophy. GeneReviews. 2000. 1993–2022. Available from: https://pubmed.ncbi.nlm.nih.gov/20301526/
4. International SMA Consortium Meeting (26–28 June 1992, Bonn, Germany)
5. International SMA Collaboration
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1. What Can RNA-Based Therapy Do for Monogenic Diseases?;Pharmaceutics;2023-01-12
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