The stony road to phe508del CFTR pharmacotherapy: smoothing the first rock
- PMID: 24973279
- DOI: 10.1016/S2213-2600(14)70136-5
The stony road to phe508del CFTR pharmacotherapy: smoothing the first rock
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A CFTR corrector (lumacaftor) and a CFTR potentiator (ivacaftor) for treatment of patients with cystic fibrosis who have a phe508del CFTR mutation: a phase 2 randomised controlled trial.Lancet Respir Med. 2014 Jul;2(7):527-38. doi: 10.1016/S2213-2600(14)70132-8. Epub 2014 Jun 24. Lancet Respir Med. 2014. PMID: 24973281 Clinical Trial.
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