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Page 1
Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia.
Thompson AA, Walters MC, Kwiatkowski J, Rasko JEJ, Ribeil JA, Hongeng S, Magrin E, Schiller GJ, Payen E, Semeraro M, Moshous D, Lefrere F, Puy H, Bourget P, Magnani A, Caccavelli L, Diana JS, Suarez F, Monpoux F, Brousse V, Poirot C, Brouzes C, Meritet JF, Pondarré C, Beuzard Y, Chrétien S, Lefebvre T, Teachey DT, Anurathapan U, Ho PJ, von Kalle C, Kletzel M, Vichinsky E, Soni S, Veres G, Negre O, Ross RW, Davidson D, Petrusich A, Sandler L, Asmal M, Hermine O, De Montalembert M, Hacein-Bey-Abina S, Blanche S, Leboulch P, Cavazzana M. Thompson AA, et al. Among authors: bourget p. N Engl J Med. 2018 Apr 19;378(16):1479-1493. doi: 10.1056/NEJMoa1705342. N Engl J Med. 2018. PMID: 29669226 Free article. Clinical Trial.
Long-term outcomes of lentiviral gene therapy for the β-hemoglobinopathies: the HGB-205 trial.
Magrin E, Semeraro M, Hebert N, Joseph L, Magnani A, Chalumeau A, Gabrion A, Roudaut C, Marouene J, Lefrere F, Diana JS, Denis A, Neven B, Funck-Brentano I, Negre O, Renolleau S, Brousse V, Kiger L, Touzot F, Poirot C, Bourget P, El Nemer W, Blanche S, Tréluyer JM, Asmal M, Walls C, Beuzard Y, Schmidt M, Hacein-Bey-Abina S, Asnafi V, Guichard I, Poirée M, Monpoux F, Touraine P, Brouzes C, de Montalembert M, Payen E, Six E, Ribeil JA, Miccio A, Bartolucci P, Leboulch P, Cavazzana M. Magrin E, et al. Among authors: bourget p. Nat Med. 2022 Jan;28(1):81-88. doi: 10.1038/s41591-021-01650-w. Epub 2022 Jan 24. Nat Med. 2022. PMID: 35075288 Clinical Trial.
Gene Therapy in a Patient with Sickle Cell Disease.
Ribeil JA, Hacein-Bey-Abina S, Payen E, Magnani A, Semeraro M, Magrin E, Caccavelli L, Neven B, Bourget P, El Nemer W, Bartolucci P, Weber L, Puy H, Meritet JF, Grevent D, Beuzard Y, Chrétien S, Lefebvre T, Ross RW, Negre O, Veres G, Sandler L, Soni S, de Montalembert M, Blanche S, Leboulch P, Cavazzana M. Ribeil JA, et al. Among authors: bourget p. N Engl J Med. 2017 Mar 2;376(9):848-855. doi: 10.1056/NEJMoa1609677. N Engl J Med. 2017. PMID: 28249145 Free article.
Haploidentical Hematopoietic Stem Cell Transplantation with Post-Transplant Cyclophosphamide for Primary Immunodeficiencies and Inherited Disorders in Children.
Neven B, Diana JS, Castelle M, Magnani A, Rosain J, Touzot F, Moreira B, Fremond ML, Briand C, Bendavid M, Levy R, Morelle G, Vincent M, Magrin E, Bourget P, Chatenoud L, Picard C, Fischer A, Moshous D, Blanche S. Neven B, et al. Among authors: bourget p. Biol Blood Marrow Transplant. 2019 Jul;25(7):1363-1373. doi: 10.1016/j.bbmt.2019.03.009. Epub 2019 Mar 12. Biol Blood Marrow Transplant. 2019. PMID: 30876929 Free article. Clinical Trial.
Midostaurin in Advanced Systemic Mastocytosis.
Chandesris MO, Damaj G, Canioni D, Brouzes C, Lhermitte L, Hanssens K, Frenzel L, Cherquaoui Z, Durieu I, Durupt S, Gyan E, Beyne-Rauzy O, Launay D, Faure C, Hamidou M, Besnard S, Diouf M, Schiffmann A, Niault M, Jeandel PY, Ranta D, Gressin R, Chantepie S, Barete S, Dubreuil P, Bourget P, Lortholary O, Hermine O; CEREMAST Study Group. Chandesris MO, et al. Among authors: bourget p. N Engl J Med. 2016 Jun 30;374(26):2605-7. doi: 10.1056/NEJMc1515403. N Engl J Med. 2016. PMID: 27355555 Clinical Trial. No abstract available.
[A medical-pharmaceutical partnership model as a contributor to the success in conditioning regimen for allogenic hematopoietic stem cell transplantation in adults: a cross-reflection on our organizations].
Bourget P, Falaschi L, Suarez F, Galland V, Blot D, Trompette C, Sibon D, Fontbrune FS, Merlette C, Vidal F, Corriol O, Giraud B, Broissand C, Clement R, Hermine O. Bourget P, et al. Bull Cancer. 2012 Jun;99(6):643-53. doi: 10.1684/bdc.2012.1589. Bull Cancer. 2012. PMID: 22645281 Free article. French.
Intracerebral gene therapy in children with mucopolysaccharidosis type IIIB syndrome: an uncontrolled phase 1/2 clinical trial.
Tardieu M, Zérah M, Gougeon ML, Ausseil J, de Bournonville S, Husson B, Zafeiriou D, Parenti G, Bourget P, Poirier B, Furlan V, Artaud C, Baugnon T, Roujeau T, Crystal RG, Meyer C, Deiva K, Heard JM. Tardieu M, et al. Among authors: bourget p. Lancet Neurol. 2017 Sep;16(9):712-720. doi: 10.1016/S1474-4422(17)30169-2. Epub 2017 Jul 14. Lancet Neurol. 2017. PMID: 28713035 Clinical Trial.
New dosing nomogram and population pharmacokinetic model for young and very young children receiving busulfan for hematopoietic stem cell transplantation conditioning.
Poinsignon V, Faivre L, Nguyen L, Neven B, Broutin S, Moshous D, Bourget P, Dufour C, Dalle JH, Galambrun C, Devictor B, Kemmel V, De Berranger E, Gandemer V, Vannier JP, Jubert C, Bondu S, Mir O, Petain A, Vassal G, Paci A. Poinsignon V, et al. Among authors: bourget p. Pediatr Blood Cancer. 2020 Oct;67(10):e28603. doi: 10.1002/pbc.28603. Epub 2020 Jul 24. Pediatr Blood Cancer. 2020. PMID: 32706505
Liquid chromatography-tandem mass spectrometry assay for therapeutic drug monitoring of the tyrosine kinase inhibitor, midostaurin, in plasma from patients with advanced systemic mastocytosis.
Bourget P, Amin A, Chandesris MO, Vidal F, Merlette C, Hirsch I, Cabaret L, Carvalhosa A, Mogenet A, Frenzel L, Damaj G, Lortholary O, Hermine O. Bourget P, et al. J Chromatogr B Analyt Technol Biomed Life Sci. 2014 Jan 1;944:175-81. doi: 10.1016/j.jchromb.2013.11.003. Epub 2013 Nov 15. J Chromatogr B Analyt Technol Biomed Life Sci. 2014. PMID: 24316764
117 results