Experimental drug shows promise for cystic fibrosis in small trial
NCT ID NCT02325362
First seen Nov 20, 2025 · Last updated May 16, 2026 · Updated 24 times
Summary
This study tested whether the drug miglustat can help fix a key protein problem in people with cystic fibrosis who have the F508del gene mutation. Sixteen adults took either miglustat or a placebo, then switched, to see if the drug improved chloride movement in their nasal passages. The goal was to see if miglustat could restore some function to the faulty CFTR protein, which is the root cause of cystic fibrosis.
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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Locations
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Assistance publique-Hôpitaux de Paris, Hôpital Cochin
Paris, 75014, France
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