Orkambi (lumacaftor/ivacaftor) - Vertex
A longitudinal multi-omic dataset of pediatric cystic fibrosis patients receiving lumacaftor/ivacaftor therapy: clinical, microbiome, inflammatory and metabolomic measurements collected over 24 months. (PubMed, Data Brief) - Oct 3, 2026 - "Microbiome data were generated from stool, sputum and throat swab samples using 16S rRNA gene sequencing, while serum metabolomics was assessed using untargeted mass spectrometry. The dataset is publicly available through SRA, MassIVE and GitHub repositories and may support future studies of longitudinal host-microbiome interactions, biomarker discovery, methodological benchmarking and comparative analyses across CFTR modulator eras." 
Journal • Cystic Fibrosis • Genetic Disorders • Immunology • Pediatrics • Pulmonary Disease • Respiratory Diseases
http://www.ncbi.nlm.nih.gov/pubmed/42825108
 
Chen Meng; Juliane Rössler; Karin Kleigrewe; Katja Hilbert; Krystyna Poplawska; Morgan Essex; Oliver Nitsche; Rebecca Luise Knoll; Sofia Kirke Forslund-Startceva; Stephan Gehring; Theda Ulrike Patricia Bartolomaeus; Ulrike Löber; Víctor Hugo Jarquín-Díaz; Virginia Rossow
 
Oct 3, 2026
 
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