Detalhe da pesquisa
1.
Betibeglogene Autotemcel Gene Therapy for Non-ß0/ß0 Genotype ß-Thalassemia.
N Engl J Med
; 386(5): 415-427, 2022 02 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-34891223
2.
Pubertal development of transfusion-dependent thalassemia patients in the era of oral chelation with deferasirox: results from the French registry.
Haematologica
; 2024 Feb 22.
Artigo
em Inglês
| MEDLINE | ID: mdl-38385265
3.
ß-Thalassemia in childhood: Current state of health in a high-income country.
Br J Haematol
; 201(2): 334-342, 2023 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-36606625
4.
Pregnancy outcome in women with transfused beta-thalassemia in France.
Ann Hematol
; 101(2): 289-296, 2022 Feb.
Artigo
em Inglês
| MEDLINE | ID: mdl-34668980
5.
Improved stenosis outcome in stroke-free sickle cell anemia children after transplantation compared to chronic transfusion.
Br J Haematol
; 193(1): 188-193, 2021 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-33216975
6.
Long-term event-free survival, chimerism and fertility outcomes in 234 patients with sickle-cell anemia younger than 30 years after myeloablative conditioning and matched-sibling transplantation in France.
Haematologica
; 105(1): 91-101, 2020 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-31097628
7.
Genotype/phenotype correlations of childhood-onset congenital sideroblastic anaemia in a European cohort.
Br J Haematol
; 187(4): 530-542, 2019 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-31338833
8.
Clinical and biological features in PIEZO1-hereditary xerocytosis and Gardos channelopathy: a retrospective series of 126 patients.
Haematologica
; 104(8): 1554-1564, 2019 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-30655378
9.
Mobilization and collection of CD34 + cells in patients with globin disorders: Providing the starting material to manufacturers of autologous gene therapies.
Transfus Apher Sci
; 63(3): 103926, 2024 Jun.
Artigo
em Inglês
| MEDLINE | ID: mdl-38670856
10.
Association of Matched Sibling Donor Hematopoietic Stem Cell Transplantation With Transcranial Doppler Velocities in Children With Sickle Cell Anemia.
JAMA
; 321(3): 266-276, 2019 01 22.
Artigo
em Inglês
| MEDLINE | ID: mdl-30667500
11.
Pediatric-onset Evans syndrome: Heterogeneous presentation and high frequency of monogenic disorders including LRBA and CTLA4 mutations.
Clin Immunol
; 188: 52-57, 2018 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-29330115
12.
Late effects after hematopoietic stem cell transplantation for ß-thalassemia major: the French national experience.
Haematologica
; 103(7): 1143-1149, 2018 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-29599204
13.
A mutation in the Gardos channel is associated with hereditary xerocytosis.
Blood
; 126(11): 1273-80, 2015 Sep 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-26148990
14.
Homozygosity for the hyperunstable hemoglobin variant Hb Agrinio (HBA2:c.89T>C) leads to severe antenatal anemia: Eight new cases in three families.
Am J Hematol
; 97(11): E393-E395, 2022 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-36052950
15.
Allogeneic/Matched Related Transplantation for ß-Thalassemia and Sickle Cell Anemia.
Adv Exp Med Biol
; 1013: 89-122, 2017.
Artigo
em Inglês
| MEDLINE | ID: mdl-29127678
16.
Mutations in TRNT1 cause congenital sideroblastic anemia with immunodeficiency, fevers, and developmental delay (SIFD).
Blood
; 124(18): 2867-71, 2014 Oct 30.
Artigo
em Inglês
| MEDLINE | ID: mdl-25193871
17.
Platelet and not erythrocyte microparticles are procoagulant in transfused thalassaemia major patients.
Br J Haematol
; 171(4): 615-24, 2015 Nov.
Artigo
em Inglês
| MEDLINE | ID: mdl-26205481
18.
A novel syndrome of congenital sideroblastic anemia, B-cell immunodeficiency, periodic fevers, and developmental delay (SIFD).
Blood
; 122(1): 112-23, 2013 Jul 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-23553769
19.
Mortality in children with sickle cell disease in mainland France from 2000 to 2015.
Haematologica
; 105(9): e440-443, 2020 09 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-33054059
20.
A genetic score for the prediction of beta-thalassemia severity.
Haematologica
; 100(4): 452-7, 2015 Apr.
Artigo
em Inglês
| MEDLINE | ID: mdl-25480500