Personalized medicine breakthrough: Lab-Grown Mini-Guts guide treatment for rare CF cases
NCT ID NCT06468527
Summary
This study tested whether a three-drug combination (Dirocaftor/Posenacaftor/Nesolicaftor) could help adults with cystic fibrosis who have rare genetic mutations not covered by existing treatments. Researchers first used patients' own lab-grown intestinal tissue samples to predict who might respond to the drugs, then tested the treatment in 41 selected participants. The main goal was to see if this personalized approach could improve lung function and validate the use of organoid screening to match patients with effective therapies.
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Contacts and locations
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Locations
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Azienda Ospedaliera Universitaria Integrata
Verona, Italy
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CHU de Nice
Nice, France
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Charité Universitätsmedizin Berlin
Berlin, Germany
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Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
Milan, Italy
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Hospital Vall d'Hebron
Barcelona, Spain
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Hospital de Santa Maria
Lisbon, Portugal
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Hôpital Larrey CHU Toulouse
Toulouse, France
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Instituto Giannina Gaslini
Genova, Italy
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Medizinische Hochschule Hannover
Hanover, Germany
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Ospedale Pediatrico Bambino Gesù
Rome, Italy
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Royal Brompton Hospital
London, United Kingdom
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Sahlgrenska University Hospital, Gothenburg CF center
Gothenburg, Sweden
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UMC Utrecht
Utrecht, Utrecht, 3584 CX, Netherlands
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UZ Leuven
Leuven, Vlaams-Brabant, Belgium
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University Hospital Southampton
Southampton, United Kingdom
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University Hospitals Birmingham NHS Foundation Trust
Birmingham, United Kingdom
Conditions
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