In vitro data can be used to accelerate the approval of drugs that target specific disease-causing mutations for additional subpopulations of patients with rare diseases such as cystic fibrosis.
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Kingwell, K. FDA OKs first in vitro route to expanded approval. Nat Rev Drug Discov 16, 591–592 (2017). https://doi.org/10.1038/nrd.2017.140
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DOI: https://doi.org/10.1038/nrd.2017.140
This article is cited by
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The impact of FDA and EMA regulatory decision-making process on the access to CFTR modulators for the treatment of cystic fibrosis
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