159 results on '"de Boeck, K"'
Search Results
2. WS09.02 Theratyping molecular defects of CFTR rare variants in patient derived rectal organoids
3. P071 Evaluation of CFTR modulator efficacy by rectal organoid morphology analysis (ROMA) indexes
4. WS04.06 Validation of rectal organoid morphology analysis (ROMA) as a novel physiological CFTR assay for diagnosis of cystic fibrosis
5. ECFS standards of care on CFTR-related disorders: Updated diagnostic criteria
6. 692 Importance of cystic fibrosis transmembrane conductance regulator messenger ribonucleic acid testing to uncover other variants in cystic fibrosis genotype that affect cystic fibrosis transmembrane conductance regulator expression
7. WS06.01 HIT-CF organoid screen with ELX-02 for people with CFTR nonsense mutations as a predictive tool for clinical response
8. P025 Assessment of CFTR modulator combinations in rectal organoids from F508del homozygous patients with cystic fibrosis
9. 660: Screening of ELX-02 readthrough effect by forskolin-induced swelling assay in CFTR nonsense mutation–bearing organoids as predictive test for clinical trial patient stratification
10. Speeding up access to new drugs for CF: Considerations for clinical trial design and delivery
11. P031 The last 10%: small molecule screening for correctors of rare CFTRprocessing mutations
12. WS09.2 Elexacaftor combinations (partially) restore CFTR function to previously drug-refractory mutations G85E and N1303K
13. P023 Characterisation of CFTR function in a patient with the F508del/ CFTRdup1–3 genotype
14. Cystic fibrosis drug trial design in the era of CFTR modulators associated with substantial clinical benefit: stakeholders’ consensus view
15. Building global development strategies for cf therapeutics during a transitional cftr modulator era
16. Use of a mobile application for self-management of pancreatic enzyme replacement therapy is associated with improved gastro-intestinal related quality of life in children with Cystic Fibrosis
17. Efficacy and safety of ataluren in patients with nonsense-mutation cystic fibrosis not receiving chronic inhaled aminoglycosides: The international, randomized, double-blind, placebo-controlled Ataluren Confirmatory Trial in Cystic Fibrosis (ACT CF)
18. WS11.3 The last 10%: small molecule screening for correctors of rare CFTR processing mutations
19. WS11.1 Response to CFTR modulators in organoids from patients with cystic fibrosis as a tool to select candidates to treatment
20. Theranostics by testing CFTR modulators in patient-derived materials: The current status and a proposal for subjects with rare CFTR mutations
21. Open label study of inhaled aztreonam for Pseudomonas eradication in children with cystic fibrosis: The ALPINE study
22. P214 Cumulative radiation exposure of radiologic imaging in patients with cystic fibrosis
23. ePS1.03 Investigation of in vitro treatment response to CFTR modulators in patients with cystic fibrosis in a cross-sectional intestinal organoid study
24. ePS5.08 MyCyFAPP project: use of a mobile application for self-management of PERT improves gastrointestinal related quality of life in children with cystic fibrosis
25. WS04-6 Development of the Rectal Organoid Morphology Analysis (ROMA) as a diagnostic test for cystic fibrosis
26. WS13-1 CFTR processing mutations cause distinct trafficking and functional defects
27. P196 Enterobacteriaceae in airway samples of preschool children with cystic fibrosis and worse outcome
28. P204 Hypogammaglobulinemia in children with cystic fibrosis
29. P021 A phase 3, open-label study of tezacaftor/ivacaftor (TEZ/IVA) therapy: interim analysis of pooled safety, and efficacy in patients homozygous for F508del-CFTR
30. WS17.4 How organoid assay results concur with the clinical phenotype in an unusual patient with S1251N/G542X
31. WS15.3 Patterns of response to lumacaftor and ivacaftor in rectal organoids
32. WS17.2 Using a highly parallel sequencing assay for CFTR genotyping in ethnically diverse European patients with CF
33. P178 Sodium status in children with CF: evaluation of fractional sodium excretion in a paediatric cohort
34. WS01.4 GLPG2222 in subjects with cystic fibrosis and the F508del/Class III mutation on stable treatment with ivacaftor: results from a phase II study (ALBATROSS)
35. WS15.6 A phase 3, open-label study of tezacaftor/ivacaftor (TEZ/IVA) therapy: interim analysis of pooled safety, and efficacy in patients heterozygous for F508del-CFTR and a residual function mutation
36. Real life practice of sweat testing in Europe
37. A European consensus for the evaluation and management of infants with an equivocal diagnosis following newborn screening for cystic fibrosis
38. EPS5.2 Genetic epidemiology of CFTR nonsense mutations across Europe
39. 341 MyCyFAPP project: assessment and validation of the PEDsQL GI symptom scale in children with CF
40. WS18.5 Evaluation of the contributions of splicing and gating defects to dysfunction of G970R-CFTR
41. WS10.2 Organoids as diagnostic test for cystic fibrosis
42. 58 Pharmacokinetics and safety of a novel CFTR corrector molecule GLPG2222 in subjects with cystic fibrosis (CF): results from a phase Ib study
43. 29 Validating the organoid model across European laboratories
44. WS13.1 QR-010, an investigational RNA therapeutic, improves CFTR activity in cystic fibrosis subjects homozygous for the F508del mutation
45. WS19.4 Predictors of long term changes in lung clearance index in children with CF
46. 227 CFCT, LCI and FEV1 in children with PCD compared to CF
47. EPS3.7 Portal hypertension in cystic fibrosis-related liver disease is a non-cirrhotic portal hypertension due to obliterative portal venopathy
48. EPS4.4 Correlation between long-term changes in LCI, FEV1 and CFCT score in children with cystic fibrosis
49. WS13.3 Efficacy and safety of ataluren in patients with nonsense-mutation cystic fibrosis not receiving chronic inhaled aminoglycosides: the international, randomized, double-blind, placebo-controlled Ataluren Confirmatory Trial in Cystic Fibrosis (ACT CF)
50. Inhaled dry powder mannitol in children with cystic fibrosis: A randomised efficacy and safety trial
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