338 results on '"Thuret, Isabelle"'
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2. Mobilization and collection of CD34 + cells in patients with globin disorders: Providing the starting material to manufacturers of autologous gene therapies
3. Recent results of hematopoietic stem cell transplantation for thalassemia with busulfan-based conditioning regimen in France: improved thalassemia free survival despite frequent mixed chimerism. A retrospective study from the Francophone Society of Stem Cell Transplantation and Cellular Therapy (SFGM-TC)
4. SARS-CoV-2 infection in patients with β-thalassemia: The French experience
5. Pregnancy outcome in women with transfused beta-thalassemia in France
6. MOBILIZATION AND COLLECTION OF CD34+ CELLS IN PATIENTS WITH GLOBIN DISORDERS: PROVIDING THE STARTING MATERIAL TO MANUFACTURERS OF AUTOLOGOUS GENE THERAPIES
7. Sustained Efficacy and Safety in Adult and Pediatric Patients with Transfusion-Dependent β-Thalassemia up to 9 Years Post Treatment with Betibeglogene Autotemcel (Beti-cel)
8. Betibeglogene Autotemcel Gene Therapy in Patients with Transfusion-Dependent, Severe Genotype β‑Thalassaemia: Results from the HGB-212 (Northstar-3) Study
9. Pediatric-onset Evans syndrome: Heterogeneous presentation and high frequency of monogenic disorders including LRBA and CTLA4 mutations
10. Allogeneic/Matched Related Transplantation for β-Thalassemia and Sickle Cell Anemia
11. Design of the DREPAGREFFE trial: A prospective controlled multicenter study evaluating the benefit of genoidentical hematopoietic stem cell transplantation over chronic transfusion in sickle cell anemia children detected to be at risk of stroke by transcranial Doppler (NCT 01340404)
12. P1451: PUBERTAL DEVELOPMENT OF TRANSFUSION DEPENDENT THALASSEMIA PATIENTS AT THE ERA OF ORAL CHELATION WITH DEFERASIROX: RESULTS OF THE FRENCH NATIONAL REGISTRY NATHALY
13. A mutation in the Gardos channel is associated with hereditary xerocytosis
14. Long-term Outcomes of 63 Patients with Transfusion-Dependent β-Thalassemia (TDT) Followed-up to 7 Years after Treatment with betibeglogene autotemcel (beti-cel) Gene Therapy (GT) and Factors Impacting Neutrophil and Platelet Engraftment
15. Long-Term Patient-Reported Outcomes Following Treatment with betibeglogene autotemcel in Patients with Transfusion-Dependent β-Thalassemia
16. Long Term Outcomes of 63 Patients with Transfusion-Dependent β-Thalassemia (TDT) Followed up to 7 Years Post-Treatment with betibeglogene autotemcel (beti-cel) Gene Therapy and Exploratory Analysis of Predictors of Successful Treatment Outcomes in Phase 3 Trials
17. Homozygosity for the hyperunstable hemoglobin variant Hb Agrinio (HBA2:c.89T>C) leads to severe antenatal anemia: Eight new cases in three families
18. A novel syndrome of congenital sideroblastic anemia, B-cell immunodeficiency, periodic fevers, and developmental delay (SIFD)
19. Post-transfusional iron overload in the haemoglobinopathies
20. French Multicenter 22-Year Experience in Stem Cell Transplantation for Beta-Thalassemia Major: Lessons and Future Directions
21. Prevalence and risk factors of the metabolic syndrome in adult survivors of childhood leukemia
22. Impact of Viable CD45 Cells Infused on Lymphocyte Subset Recovery after Unrelated Cord Blood Transplantation in Children
23. Efficacy and Safety of Betibeglogene Autotemcel (beti-cel) Gene Therapy in 63 Patients with Transfusion-Dependent β-Thalassemia (TDT): 7-Year Post-Infusion Follow-up of Phase 1/2 and Phase 3 Studies
24. β‐Thalassemia in childhood: Current state of health in a high‐income country.
25. Betibeglogene Autotemcel Gene Therapy for Non–β0/β0 Genotype β-Thalassemia
26. 122 - Safety and Efficacy Outcomes in Pediatric Patients with Transfusion-Dependent β-Thalassemia (TDT) Receiving Betibeglogene Autotemcel (beti-cel; LentiGlobin for β-thalassemia) Gene Therapy in the Phase 3 Hgb-207 (Northstar-2) and Hgb-212 (Northstar-3) Studies
27. Platelet and not erythrocyte microparticles are procoagulant in transfused thalassaemia major patients
28. Restoring Iron Homeostasis in Pts Who Achieved Transfusion Independence after Treatment with Betibeglogene Autotemcel Gene Therapy: Results from up to 7 Years of Follow-up
29. Improvement in Health-Related Quality of Life Following Treatment with Betibeglogene Autotemcel in Patients with Transfusion-Dependent β-Thalassemia Enrolled in Phase 3 Studies
30. Les alternatives médicamenteuses à la TF : anciennes et nouvelles molécules
31. Place de la thérapie génique dans les thalassémies
32. Pregnancy outcome in women with transfused beta-thalassemia in France
33. Rare Duplication or Deletion of Exons 6, 7 and 8 in CYBB Leading to X-Linked Chronic Granulomatous Disease in Two Patients from Different Families
34. Neonatal screening for sickle cell disease in France: evaluation of the selective process
35. Response of Patients with Transfusion-Dependent β-Thalassemia (TDT) to Betibeglogene Autotemcel (beti-cel; LentiGlobin for β-Thalassemia) Gene Therapy Based on HBB Genotype and Disease Genetic Modifiers
36. Favorable Outcomes in Pediatric Patients in the Phase 3 Hgb-207 (Northstar-2) and Hgb-212 (Northstar-3) Studies of Betibeglogene Autotemcel Gene Therapy for the Treatment of Transfusion-Dependent β-Thalassemia
37. Peripheral blood cells chimerism after unrelated cord blood transplantation in children: kinetics, predictive factors and impact on post-transplant outcome
38. Improvement in Iron Burden in Patients with Transfusion-Dependent β-Thalassemia (TDT) Treated with Betibeglogene Autotemcel (Beti-cel) Gene Therapy: Up to 9 Years of Follow-up
39. Sustained Efficacy, Safety, and Improved Quality of Life in Adult and Pediatric Patients with Transfusion-Dependent β-Thalassemia up to 9 Years Post Treatment with Betibeglogene Autotemcel (Beti-cel)
40. Long-term results of related myeloablative stem-cell transplantation to cure sickle cell disease
41. Safety and Efficacy Outcomes in Pediatric Patients with Transfusion-Dependent β-Thalassemia (TDT) Receiving Betibeglogene Autotemcel (beti-cel; LentiGlobin for β-thalassemia) Gene Therapy in the Phase 3 Hgb-207 (Northstar-2) and Hgb-212 (Northstar-3) Studies
42. Efficacy and Safety of Betibeglogene Autotemcel (beti-cel; LentiGlobin for β-thalassemia) Gene Therapy in 60 Patients with Transfusion-Dependent β-Thalassemia (TDT) Followed for up to 6 Years Post-Infusion
43. Early versus deferred antiretroviral multidrug therapy in infants infected with HIV type 1
44. A phase 3 study of deferasirox (ICL670), a once-daily oral iron chelator, in patients with β-thalassemia
45. Lopinavir/ritonavir population pharmacokinetics in neonates and infants
46. The Clinical Severity of Alpha-2 Globin Gene Variants: Homozygosity for Hb Agrinio (HBA2: c.89T>C) Leads to Severe Antenatal Anemia, about 8 Cases in 3 Families
47. Mortality in children with sickle cell disease in mainland France from 2000 to 2015
48. Interim Results from the Phase 3 Hgb-207 (Northstar-2) and Hgb-212 (Northstar-3) Studies of Betibeglogene Autotemcel Gene Therapy (LentiGlobin) for the Treatment of Transfusion-Dependent β-Thalassemia
49. Overview of Sickle-cell disease management in the Pediatric Hematology and Oncology Center of Rabat (Morocco)
50. Raltegravir, etravirine and r-darunavir combination in adolescents with multidrug-resistant virus
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