Detalhe da pesquisa
1.
Outcomes of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome.
Blood
; 142(15): 1281-1296, 2023 10 12.
Artigo
Inglês
| MEDLINE | ID: mdl-37478401
2.
Post-Transcriptional Genetic Silencing of BCL11A to Treat Sickle Cell Disease.
N Engl J Med
; 384(3): 205-215, 2021 01 21.
Artigo
Inglês
| MEDLINE | ID: mdl-33283990
3.
Risk factors and outcomes of melanoma in children and adolescents: A retrospective multicenter study.
J Am Acad Dermatol
; 90(4): 716-726, 2024 Apr.
Artigo
Inglês
| MEDLINE | ID: mdl-38040338
4.
Clinical and biological features prognostic of survival after relapse or progression of INRGSS stage MS pattern neuroblastoma: A report from the International Neuroblastoma Risk Group (INRG) project.
Pediatr Blood Cancer
; 70(2): e30054, 2023 02.
Artigo
Inglês
| MEDLINE | ID: mdl-36316811
5.
Phase 1 study of cabozantinib in combination with topotecan-cyclophosphamide for patients with relapsed Ewing sarcoma or osteosarcoma.
Pediatr Blood Cancer
; 70(12): e30681, 2023 Dec.
Artigo
Inglês
| MEDLINE | ID: mdl-37715723
6.
Impact of diagnostic and end-of-induction Curie scores with tandem high-dose chemotherapy and autologous transplants for metastatic high-risk neuroblastoma: A report from the Children's Oncology Group.
Pediatr Blood Cancer
; 70(8): e30418, 2023 Aug.
Artigo
Inglês
| MEDLINE | ID: mdl-37199022
7.
The pyruvate kinase (PK) to hexokinase enzyme activity ratio and erythrocyte PK protein level in the diagnosis and phenotype of PK deficiency.
Br J Haematol
; 192(6): 1092-1096, 2021 03.
Artigo
Inglês
| MEDLINE | ID: mdl-32463523
8.
Pyruvate kinase deficiency in children.
Pediatr Blood Cancer
; 68(9): e29148, 2021 09.
Artigo
Inglês
| MEDLINE | ID: mdl-34125488
9.
Phase 1 study of sorafenib and irinotecan in pediatric patients with relapsed or refractory solid tumors.
Pediatr Blood Cancer
; 68(11): e29282, 2021 11.
Artigo
Inglês
| MEDLINE | ID: mdl-34383370
10.
A nomogram of clinical and biologic factors to predict survival in children newly diagnosed with high-risk neuroblastoma: An International Neuroblastoma Risk Group project.
Pediatr Blood Cancer
; 68(3): e28794, 2021 03.
Artigo
Inglês
| MEDLINE | ID: mdl-33205902
11.
A POETIC Phase II study of continuous oral everolimus in recurrent, radiographically progressive pediatric low-grade glioma.
Pediatr Blood Cancer
; 68(2): e28787, 2021 02.
Artigo
Inglês
| MEDLINE | ID: mdl-33140540
12.
Comparison of Outcomes of Myeloablative Allogeneic Stem Cell Transplantation for Pediatric Patients with Bone Marrow Failure, Myelodysplastic Syndrome and Acute Myeloid Leukemia with and without Germline GATA2 Mutations.
Biol Blood Marrow Transplant
; 26(6): 1124-1130, 2020 06.
Artigo
Inglês
| MEDLINE | ID: mdl-32088370
13.
Clinical spectrum of pyruvate kinase deficiency: data from the Pyruvate Kinase Deficiency Natural History Study.
Blood
; 131(20): 2183-2192, 2018 05 17.
Artigo
Inglês
| MEDLINE | ID: mdl-29549173
14.
Genotype-phenotype correlation and molecular heterogeneity in pyruvate kinase deficiency.
Am J Hematol
; 95(5): 472-482, 2020 05.
Artigo
Inglês
| MEDLINE | ID: mdl-32043619
15.
The prognostic strength of serum LDH and serum ferritin in children with neuroblastoma: A report from the International Neuroblastoma Risk Group (INRG) project.
Pediatr Blood Cancer
; 67(8): e28359, 2020 08.
Artigo
Inglês
| MEDLINE | ID: mdl-32472746
16.
A retrospective multicenter study of fatal pediatric melanoma.
J Am Acad Dermatol
; 83(5): 1274-1281, 2020 Nov.
Artigo
Inglês
| MEDLINE | ID: mdl-32622142
17.
Correction: Detection of circulating tumour DNA is associated with inferior outcomes in Ewing sarcoma and osteosarcoma: a report from the Children's Oncology Group.
Br J Cancer
; 120(8): 869, 2019 Apr.
Artigo
Inglês
| MEDLINE | ID: mdl-30880335
18.
Risk Factors and Outcomes of Nonmelanoma Skin Cancer in Children and Young Adults.
J Pediatr
; 211: 152-158, 2019 08.
Artigo
Inglês
| MEDLINE | ID: mdl-31103258
19.
Comprehensive evaluation of context dependence of the prognostic impact of MYCN amplification in neuroblastoma: A report from the International Neuroblastoma Risk Group (INRG) project.
Pediatr Blood Cancer
; 66(8): e27819, 2019 08.
Artigo
Inglês
| MEDLINE | ID: mdl-31115156
20.
The challenge of defining "ultra-high-risk" neuroblastoma.
Pediatr Blood Cancer
; 66(4): e27556, 2019 04.
Artigo
Inglês
| MEDLINE | ID: mdl-30479064