Two decades on from the discovery of the cystic fibrosis gene, the first disease-modifying drug stands at the gateway to approval.
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Opar, A. Excitement mounts for first disease-modifying cystic fibrosis drugs. Nat Rev Drug Discov 10, 479–480 (2011). https://doi.org/10.1038/nrd3488
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DOI: https://doi.org/10.1038/nrd3488
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