54 results on '"Cystic fibrosis -- Care and treatment -- Genetic aspects"'
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2. European Commission approves Vertex Pharma's Kaftrio in combo with ivacaftor to treat children with cystic fibrosis ages 2 through 5
3. Prime Medicine Receives Up to $15 Mn from Cystic Fibrosis Foundation to Advance Hotspot and PASSIGEtm Prime Editors for Cystic Fibrosis
4. Benchmarking organ-specific responses to therapies in tissues differentiated from Cystic Fibrosis patient derived iPSCs
5. Small-molecule eRF3a degraders rescue CFTR nonsense mutations by promoting premature termination codon readthrough
6. Research from Utrecht University Reveals New Findings on Molecular Science (Redefining Hypo- and Hyper-Responding Phenotypes of CFTR Mutants for Understanding and Therapy)
7. Redefining hypo- and hyper-responding phenotypes of CFTR mutants for understanding and therapy
8. UI researchers reveal possible new therapy for people who have cystic fibrosis - The Daily Iowan
9. Gene therapy could offer an inclusive cure for cystic fibrosis
10. Epigenetic and transcriptional plasticity drive inflammation in Cystic Fibrosis (Updated October 12, 2023)
11. University of Naples Researchers Yield New Study Findings on Lung Diseases and Conditions (Elexacaftor/Tezacaftor/Ivacaftor in Patients with Cystic Fibrosis Homozygous for the * * F508del* * Mutation and Advanced Lung Disease: A 48-Week ...)
12. Macrolide therapy in Pseudomonas aeruginosa infections causes uL4 ribosomal protein mutations leading to high-level resistance
13. Researchers at Cold Spring Harbor Laboratory Target Antisense Technology (Exon-skipping Antisense Oligonucleotides for Cystic Fibrosis Therapy)
14. EMA committee recommends approval of Vertex's Kaftrio in combo with ivacaftor in children with CF ages 6 through 11 years old
15. Put to the test: Organoid-based testing becomes a clinical tool
16. Health Canada accepts for priority review Vertex Pharma's sNDA for PrKalydeco to treat cystic fibrosis in patients from 4 months to 18 years of age
17. ReCode Therapeutics Announces Strategic Investment from the Cystic Fibrosis Foundation to Accelerate Development of Novel mRNA Therapy
18. Data on Cystic Fibrosis Detailed by Researchers at Hebrew University of Jerusalem (Disease Severity of People With Cystic Fibrosis Carrying Residual Function Mutations: Data From the Ecfs Patient Registry)
19. Vertex receives European approval for Symkevi with Kalydeco for eligible children with cystic fibrosis ages 6-11 years
20. EMA validates Vertex's type II variation MAA for Kaftrio in combo with ivacaftor to treat CF in patients with one copy of F508del mutation
21. Researchers at Haga Teaching Hospital Target Genistein Therapy (Clinical effects of the three CFTR potentiator treatments curcumin, genistein and ivacaftor in patients with the CFTR-S1251N gating mutation)
22. Study Data from University of Naples Federico II Update Knowledge of Cystic Fibrosis (Cystic Fibrosis Patients with F508del/Minimal Function Genotype: Laboratory and Nutritional Evaluations after One Year of Elexacaftor/Tezacaftor/Ivacaftor ...)
23. Shandong First Medical University Researchers Highlight Recent Research in Gene Therapy (Gene therapy for cystic fibrosis: Challenges and prospects)
24. European Commission Approves KAFTRIO[R] (ivacaftor/tezacaftor/elexacaftor) in Combination With Ivacaftor to Treat Children With Cystic Fibrosis Ages 6 to 11 Years
25. Kaftrio Treated Severe CF Without Confirmed Secondary CFTR Mutation
26. New Hope For Patients Living With Cystic Fibrosis After Scientists Unveil Therapy
27. European Commission Approves Kaftrio in Combination with Ivacaftor to Treat Children with Cystic Fibrosis Ages 6 to 11 Years
28. CF Foundation Invests Up to $8.4M in SpliSense for the Development of Potential Rare Mutation Therapy
29. CF Foundation Invests Up to $8.4M in SpliSense for the Development of Potential Rare Mutation Therapy
30. CF FOUNDATION INVESTS UP TO $8.4M IN SPLISENSE FOR THE DEVELOPMENT OF POTENTIAL RARE MUTATION THERAPY
31. Vertex Announces European Commission Approval for KAFTRIO(r) (ivacaftor|tezacaftor|elexacaftor) in Combination With Ivacaftor to Treat Cystic Fibrosis Patients 12 Years and Older With At Least One F508del Mutation in the CFTR gene
32. How much protein function needs to be restored in cystic fibrosis?
33. Vertex Receives CHMP Positive Opinion for Kaftrio in Combination with Ivacaftor in Children with Cystic Fibrosis Ages 6 Through 11
34. Vertex Announces European Commission Approval for SYMKEVI(r) (tezacaftor|ivacaftor) With KALYDECO(r) (ivacaftor) for Eligible Children With Cystic Fibrosis Ages 6-11 Years
35. Vertex Announces European Commission Approval for SYMKEVI[R] (tezacaftor/ivacaftor) With KALYDECO[R] (ivacaftor) for Eligible Children With Cystic Fibrosis Ages 6-11 Years
36. Vertex Announces European Commission Approval for KALYDECO[R] (ivacaftor) as First and Only CFTR Modulator to Treat Eligible Infants With Cystic Fibrosis as Early as Four Months of Age
37. Vertex Announces European Commission Approval for KALYDECO (ivacaftor) for Children and Adolescents With Cystic Fibrosis Between the Ages of 6 Months and 18 Years With the R117H Mutation in the CFTR Gene
38. Vertex Announces European Commission Approval for KALYDECO(r) (ivacaftor) for Children and Adolescents With Cystic Fibrosis Between the Ages of 6 Months and 18 Years With the R117H Mutation in the CFTR Gene
39. Vertex Announces European Commission Approval for KALYDECO[R] (ivacaftor) for Children and Adolescents With Cystic Fibrosis Between the Ages of 6 Months and 18 Years With the R117H Mutation in the CFTR Gene
40. Vertex Receives European CHMP Positive Opinion for KALYDECO[R] (ivacaftor) for Children and Adolescents With Cystic Fibrosis Between the Ages 6 Months and 18 Years With the R117H Mutation in the CFTR Gene
41. Gene-Specific Nonsense-Mediated mRNA Decay Targeting for Cystic Fibrosis Therapy
42. Investigators at New York University (NYU) Report Findings in Azoospermia (How Do We Counsel Men With Obstructive Azoospermia Due To Cf Mutations?-a Review of Treatment Options and Outcomes)
43. PTC Therapeutics completes enrolment of Phase III trial of ataluren in patients with cystic fibrosis
44. PTC Therapeutics Completes Enrollment of Phase 3 Trial of Ataluren in Patients with Cystic Fibrosis
45. Copernicus to Receive Increased Support from Cystic Fibrosis Foundation Therapeutics to Further Development of its Non-Viral Gene Therapy for Cystic Fibrosis
46. Panel Permits Use of Genes In Treating Cystic Fibrosis
47. Potential Therapy Is Found for Cystic Fibrosis
48. Computer-designed molecules point to new therapy for cystic fibrosis
49. PTC Therapeutics Completes Enrollment of Phase 3 Trial of Ataluren in Patients with Cystic Fibrosis
50. PTC Therapeutics Initiates Registration-Directed Phase 3 Trial of Ataluren in Cystic Fibrosis
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