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1. Intensive chemotherapy followed by autologous stem cell transplantation in primary central nervous system lymphomas (PCNSLs). Therapeutic outcomes in real life—experience of the French Network

2. Long-term outcomes of lentiviral gene therapy for the β-hemoglobinopathies: the HGB-205 trial

3. Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia

4. Haploidentical Hematopoietic Stem Cell Transplantation with Post-Transplant Cyclophosphamide for Primary Immunodeficiencies and Inherited Disorders in Children

6. Intracerebral gene therapy in children with mucopolysaccharidosis type IIIB syndrome: an uncontrolled phase 1/2 clinical trial

10. Design and Development of a User-Friendly Motorized Apparatus for the Filling of Portable Infusion Pumps by Hospital and Homecare Health Workers

12. Supplement to: Gene thereapy in a patient with sickle cell disease.

13. Gene Therapy in a Patient with Sickle Cell Disease: Brief Report

14. Modèle de coopération médico-pharmaceutique comme contributeur au succès du conditionnement de la greffe allogénique de cellules souches hématopoïétiques chez l’adulte : un regard croisé sur nos organisations

21. New dosing nomogram and population pharmacokinetic model for young and very young children receiving busulfan for hematopoietic stem cell transplantation conditioning

29. Results from the Completed Hgb-205 Trial of Lentiglobin for β-Thalassemia and Lentiglobin for Sickle Cell Disease Gene Therapy

30. Intracerebral administration of rAAV2/5hNAGLU vector in children with MPS IIIB: results at 30 months of a phase I/II trial

31. Abstract B57: Effects of MEK inhibition alone or in combination with PI3K-mTOR pathway inhibitors in pancreatic ductal adenocarcinoma in vitro and on an innovative ex vivo fresh tumor tissue culture model

32. Update from the Hgb-205 Phase 1/2 Clinical Study of Lentiglobin Gene Therapy: Sustained Clinical Benefit in Severe Hemoglobinopathies

33. Midostaurin in Advanced Systemic Mastocytosis

34. 279. Clinical Outcomes of Gene Therapy with BB305 Lentiviral Vector for Sickle Cell Disease and β-Thalassemia

36. Reiterated Therapeutic Drug Monitoring (TDM) Dosing to Significantly Improve the Control of Exposure to IV Busulfan in Infants and Older Children Undergoing Hematopoietic Stem-Cell Transplantation (HSCT)

37. Outcomes of Gene Therapy for Severe Sickle Disease and Beta-Thalassemia Major Via Transplantation of Autologous Hematopoietic Stem Cells Transduced Ex Vivo with a Lentiviral Beta AT87Q-Globin Vector

39. Abstract 2677: Decrease in phospho-S6 expression under MEK inhibitor (MEKi) treatment as a potential predictive biomarker of response to MEKi alone or in combination with PI3K-mTOR pathway inhibitor in pancreatic adenocarcinoma in vitro and ex vivo models

40. Decreased pS6 expression under MEK inhibitor (MEKi) correlates with response to MEKi alone or combined with PI3Ki-mTOR inhibitor (PI3Ki-mTORi) in pancreatic adenocarcinoma (PAC) in vitro and ex vivo models

41. Correlation of decreased pS6 expression under MEK inhibitor (MEKi) with response to MEKi alone or combined with PI3K-mTOR inhibitor (PI3Ki-mTORi) in pancreatic adenocarcinoma (PAC) in vitro and ex vivo models.

42. Sunitinib as second-line treatment in patients with advanced intrahepatic cholangiocarcinoma (SUN-CK phase II trial): Safety, efficacy, and updated translational results.

44. Longer Term Follow-up on the First Patients with Severe Hemoglobinopathies Treated with Lentiglobin Gene Therapy

45. Treatment of Advanced Systemic Mastocytosis with PKC412: The French Compassionate Use Programme Experience and Historical Comparison

46. Pharmacokinetics/Pharmacodynamic Relationship in Busulfan Conditioning Regimen: Results from a Large Pediatric Cohort Undergoing Hematopoietic Stem-Cell Transplantation

47. Mechanical performances of elastomers used in diffusers

49. Development and Validation of a Sensitive Liquid Chromatography-Tandem Mass Spectrometry (LC-MS/MS) Assay for the Simultaneous Quantification of Midostaurin (PKC412) and Its Two Major Metabolites: A Suitable Tool for the Monitoring of the Drug in Patients Suffering From Advanced Systemic Mastocytosis

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