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Miscellaneous
Cystic fibrosis: a novel therapeutic angle or a false dawn
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Worldwide, approximately 10% of patients with cystic fibrosis have premature stop codons in the mRNA for the CFTR gene. These result in phenotypically severe variants of cystic fibrosis. PTC124 is a small molecule available as an oral preparation which allows the ribosome to selectively ignore these mutations and hence produce the functioning protein.
This prospective phase II trial recruited …