Detalhe da pesquisa
1.
Sickle Cell Disease: From Genetics to Curative Approaches.
Annu Rev Genomics Hum Genet
; 24: 255-275, 2023 08 25.
Artigo
em Inglês
| MEDLINE | ID: mdl-37624668
2.
Late-onset enteric virus infection associated with hepatitis (EVAH) in transplanted SCID patients.
J Allergy Clin Immunol
; 151(6): 1634-1645, 2023 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-36638922
3.
Pediatric Tumors and Developmental Anomalies: A French Nationwide Cohort Study.
J Pediatr
; 259: 113451, 2023 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-37169337
4.
Saliva for molecular detection of SARS-CoV-2 in pre-school and school-age children.
Environ Microbiol
; 24(10): 4725-4737, 2022 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-36065993
5.
Seletalisib for Activated PI3Kδ Syndromes: Open-Label Phase 1b and Extension Studies.
J Immunol
; 205(11): 2979-2987, 2020 12 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-33115853
6.
Pharmacokinetics of tranexamic acid after intravenous, intramuscular, and oral routes: a prospective, randomised, crossover trial in healthy volunteers.
Br J Anaesth
; 128(3): 465-472, 2022 Mar.
Artigo
em Inglês
| MEDLINE | ID: mdl-34998508
7.
Gene Therapy in Patients with Transfusion-Dependent ß-Thalassemia.
N Engl J Med
; 378(16): 1479-1493, 2018 04 19.
Artigo
em Inglês
| MEDLINE | ID: mdl-29669226
8.
Gene Therapy in a Patient with Sickle Cell Disease.
N Engl J Med
; 376(9): 848-855, 2017 03 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-28249145
9.
Cell-Cycle Arrest Biomarkers: Usefulness for Cardiac Surgery-Related Acute Kidney Injury in Neonates and Infants.
Pediatr Crit Care Med
; 21(6): 563-570, 2020 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-32195906
10.
Urinary Exosomes of Patients with Cystic Fibrosis Unravel CFTR-Related Renal Disease.
Int J Mol Sci
; 21(18)2020 Sep 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-32927759
11.
Sotatercept, a novel transforming growth factor ß ligand trap, improves anemia in ß-thalassemia: a phase II, open-label, dose-finding study.
Haematologica
; 104(3): 477-484, 2019 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-30337358
12.
eIF2α phosphorylation as a biomarker of immunogenic cell death.
Semin Cancer Biol
; 33: 86-92, 2015 Aug.
Artigo
em Inglês
| MEDLINE | ID: mdl-25749194
13.
Reverse-Transcriptase Inhibitors in the AicardiGoutières Syndrome
N Engl J Med
; 379(23): 2275-7, 2018 12 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-30566312
14.
Lumacaftor/Ivacaftor Population Pharmacokinetics in Pediatric Patients with Cystic Fibrosis: A First Step Toward Personalized Therapy.
Clin Pharmacokinet
; 63(3): 333-342, 2024 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-38310629
15.
Analysis of the impact of the SARS-CoV-2 infection on the pediatric population hospitalized during the pandemic in the Greater Paris University Hospitals.
Front Pediatr
; 11: 1044352, 2023.
Artigo
em Inglês
| MEDLINE | ID: mdl-36923274
16.
Long-term neurological and neurodevelopmental outcome of neonatal listeriosis in France: a prospective, matched, observational cohort study.
Lancet Child Adolesc Health
; 7(12): 875-885, 2023 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-37871603
17.
Severe hematopoietic stem cell inflammation compromises chronic granulomatous disease gene therapy.
Cell Rep Med
; 4(2): 100919, 2023 02 21.
Artigo
em Inglês
| MEDLINE | ID: mdl-36706754
18.
Long-term outcomes of lentiviral gene therapy for the ß-hemoglobinopathies: the HGB-205 trial.
Nat Med
; 28(1): 81-88, 2022 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-35075288
19.
Clinical Validation of a Volumetric Absorptive Micro-Sampling Device for Pharmacokinetic Studies With Tranexamic Acid.
Front Pharmacol
; 12: 764379, 2021.
Artigo
em Inglês
| MEDLINE | ID: mdl-34887763
20.
Sustained remission after haploidentical bone marrow transplantation in a child with refractory systemic juvenile idiopathic arthritis.
Pediatr Rheumatol Online J
; 19(1): 27, 2021 Mar 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-33712044