754 results on '"KEREM, E."'
Search Results
2. ECFS standards of care on CFTR-related disorders: Towards a comprehensive program for affected individuals
3. Stenotrophomonas maltophilia outbreak originating from a pull-out faucet in a pediatric intensive care unit in Turkey: Insights from clinical records and molecular typing
4. Impact of gut microbiota on liver transplantation
5. WS06.02 First-in-human clinical trial with SPL84, an ASO for treatment of cystic fibrosis patients carrying the 3849 +10 Kb C -> T mutation
6. WS06.06 Safety and efficacy of a nebulized phage cocktail in cystic fibrosis patients with chronic Pseudomonas aeruginosa pulmonary infection: a phase 1b/2a randomized, double-blind, placebo-controlled study
7. WS06.05 Long-term outcomes in people with CF lacking early spirometry response to elexacaftor/tezacaftor/ivacaftor therapy
8. Cystic fibrosis drug trial design in the era of CFTR modulators associated with substantial clinical benefit: stakeholders’ consensus view
9. 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)
10. Stenotrophomonas maltophilia outbreak originating from a pull-out faucet in a pediatric intensive care unit in Turkey: Insights from clinical records and molecular typing
11. Exceptionally active and stable catalysts for CO2 reforming of glycerol to syngas
12. 116 First-in-human clinical trial with SPL84, an antisense oligonucleotide for treatment of people with cystic fibrosis with the 3849 +10 Kb C - > T mutation
13. 126 A phase 1b/2a randomized, double-blind, placebo-controlled, multicenter study evaluating nebulized phage therapy in people with cystic fibrosis with chronic Pseudomonas aeruginosa pulmonary infection
14. 290 Extending the success of elexacaftor-tezacaftor-ivacaftor to rare mutations—insights from CFTR structure and modulator binding
15. P077 Clinical and functional efficacy of elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis carrying the N1303K mutation
16. P095 A phase 1b/2a randomized, double-blind, placebo-controlled, multicentre study evaluating nebulized phage therapy in cystic fibrosis subjects with chronic Pseudomonas aeruginosa pulmonary infection
17. WS09.01 Extending the success of Trikafta to rare mutations - insights from CFTR structure and modulator binding
18. 615 Antisense oligonucleotide-based drug development for people with cystic fibrosis carrying the 2789+5 G-to-A splicing mutation
19. 619 Antisense oligonucleotide splicing modulation as a novel cystic fibrosis therapeutic approach for the W1282X nonsense mutation
20. 620 Antisense oligonucleotide splicing modulation as a novel cystic fibrosis therapeutic approach for the N1303 K nonsense mutation
21. 133 Clinical response to elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis with the N1303K and non-modulator responsive mutation
22. P029 Antisense oligonucleotide-based drug development for cystic fibrosis patients carrying the 2789+5 G-to-A splicing mutation
23. P028 Antisense oligonucleotide splicing modulation as a novel cystic fibrosis (CF) therapeutic approach for the W1282X nonsense mutation
24. P088 Ventilation inhomogeneity in patients with cystic fibrosis and pancreatic sufficiency and insufficiency
25. Recommendations for the classification of diseases as CFTR-related disorders
26. 601: Antisense-oligonucleotide-splicing modulation as a novel CF therapeutic approach for W1282X mutation
27. 166 Design of a Phase 2 study in 3849 +10 Kb C->T people with cystic fibrosis to evaluate safety, tolerability, pharmacokinetics, and preliminary efficacy of SPL84.
28. Speeding up access to new drugs for CF: Considerations for clinical trial design and delivery
29. The Catalytic Enantioselective Stetter Reaction
30. Precious Metal-Free LaMnO
31. Antisense oligonucleotide-based drug development for Cystic Fibrosis patients carrying the 3849+10 kb C-to-T splicing mutation
32. Global Pediatric Pulmonology Alliance (GPPA) proposal for COVID-19 vaccination in children.
33. Consensus on the use and interpretation of cystic fibrosis mutation analysis in clinical practice
34. P052 Efficacy of elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis with normal/near normal FEV1 (≥70%)
35. WS03.01 Differences in disease severity among different residual function mutations: data from the ECFS Patient Registry
36. Chest computed tomography outcomes in a randomized clinical trial in cystic fibrosis: Lessons learned from the first ataluren phase 3 study
37. Global Pediatric Pulmonology Alliance recommendation to strengthen prevention of pediatric seasonal influenza under COVID-19 pandemic.
38. Global Pediatric Pulmonology Alliance recommendation to strengthen prevention of pediatric seasonal influenza under COVID-19 pandemic
39. Chest computed tomography outcomes in a randomized clinical trial in cystic fibrosis: Lessons learned from the first ataluren phase 3 study
40. Precious Metal-Free LaMnO3 Perovskite Catalyst with an Optimized Nanostructure for Aerobic C–H Bond Activation Reactions: Alkylarene Oxidation and Naphthol Dimerization
41. P034 Novel insights into the therapeutic potential of antisense oligonucleotides as splicing modulators in respiratory and intestinal patient-derived model systems
42. Systemic inflammatory mediators and cystic fibrosis genotype
43. Serum CA 19–9 levels as a diagnostic marker in cystic fibrosis patients with borderline sweat tests
44. DIFFERENCES IN DISEASE EXPRESSION BETWEEN CF WITH PANCREATIC INSUFFICIENCY, PANCREATIC SUFFICIENCY AND PRIMARY CILIARY DYSKINESIA: 373
45. NATURAL HISTORY OF CYSTIC FIBROSIS IN PATIENTS WITH NONSENSE-MUTATION-MEDIATED DISEASE: 252
46. RESULTS OF THE PHASE 3 STUDY OF ATALUREN IN NONSENSE MUTATION CYSTIC FIBROSIS (NMCF): 193★
47. SURVIVAL OF CYSTIC FIBROSIS - ASSOCIATED BACTERIA IN HYPERTONIC SALINE SOLUTIONS USED IN OUT-PATIENT SETTINGS: 282
48. PRETREATMENT DATA FROM PHASE 3 STUDY OF ATALUREN DOCUMENT SIGNIFICANT DISEASE BURDEN IN A SUBPOPULATION OF PATIENTS WITH NONSENSE MUTATION CYSTIC FIBROSIS: 232
49. COMPARISON OF NASAL POTENTIAL DIFFERENCE AND INTESTINAL CURRENT MEASUREMENTS AS POTENTIAL THERAPEUTIC ENDPOINT MEASURES IN CYSTIC FIBROSIS: 217
50. Whole Exome Sequencing Accuracy in the Diagnosis of Primary Ciliary Dyskinesia
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