Correlation between patient-derived intestinal organoids and clinical responses to CFTR modulators in people with cystic fibrosis homozygous for F508del

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Journal of cystic fibrosis - ISSN 1569-1993-24:5 (2025) p. 939-945
Auteur(s)
    Noelia Rodriguez Mier, Senne Cuyx, Kris De Boeck, Marijke Proesmans, Mieke Boon, Lieven Dupont, Kaline Arnauts, Elke De Wachter, Stephanie Van Biervliet, Monique Lequesne, Vicky Nowe, Hedwige Boboli, Anabela Santo Ramalho, Francois Vermeulen, Linda Boulanger, Georges Casimir, Benedicte Demeyere, Danny De Looze, Anja Delporte, Isabelle Etienne, Laurence Hanssens, Christiane Knoop, Ann Raman, Dirk Staessen, Eva Van Braeckel, Kim van Hoorenbeeck, Eef Vanderhelst, Stijn Verhulst, Stefanie Vincken

High-resolution mass spectrometry method for targeted screening and monitoring of Fabry, Gaucher and ASMD using dried blood spots and capitainers : impact of sample matrix on measurement results

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International journal of molecular sciences - ISSN 1661-6596-26:15 (2025) p. 1-18