Search details
1.
Outcomes of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome.
Blood
; 142(15): 1281-1296, 2023 10 12.
Article
in English
| 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.
Article
in English
| MEDLINE | ID: mdl-33283990
3.
Long-term follow-up of patients with intermediate-risk neuroblastoma treated with response- and biology-based therapy: A report from the Children's Oncology Group study ANBL0531.
Pediatr Blood Cancer
; : e31089, 2024 May 31.
Article
in English
| MEDLINE | ID: mdl-38822537
4.
Risk factors and outcomes of melanoma in children and adolescents: A retrospective multicenter study.
J Am Acad Dermatol
; 90(4): 716-726, 2024 Apr.
Article
in English
| MEDLINE | ID: mdl-38040338
5.
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.
Article
in English
| MEDLINE | ID: mdl-36316811
6.
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.
Article
in English
| MEDLINE | ID: mdl-37715723
7.
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.
Article
in English
| MEDLINE | ID: mdl-37199022
8.
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.
Article
in English
| MEDLINE | ID: mdl-32463523
9.
Pyruvate kinase deficiency in children.
Pediatr Blood Cancer
; 68(9): e29148, 2021 09.
Article
in English
| MEDLINE | ID: mdl-34125488
10.
Phase 1 study of sorafenib and irinotecan in pediatric patients with relapsed or refractory solid tumors.
Pediatr Blood Cancer
; 68(11): e29282, 2021 11.
Article
in English
| MEDLINE | ID: mdl-34383370
11.
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.
Article
in English
| MEDLINE | ID: mdl-33205902
12.
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.
Article
in English
| MEDLINE | ID: mdl-33140540
13.
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.
Article
in English
| MEDLINE | ID: mdl-32088370
14.
Clinical spectrum of pyruvate kinase deficiency: data from the Pyruvate Kinase Deficiency Natural History Study.
Blood
; 131(20): 2183-2192, 2018 05 17.
Article
in English
| MEDLINE | ID: mdl-29549173
15.
Genotype-phenotype correlation and molecular heterogeneity in pyruvate kinase deficiency.
Am J Hematol
; 95(5): 472-482, 2020 05.
Article
in English
| MEDLINE | ID: mdl-32043619
16.
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.
Article
in English
| MEDLINE | ID: mdl-32472746
17.
A retrospective multicenter study of fatal pediatric melanoma.
J Am Acad Dermatol
; 83(5): 1274-1281, 2020 Nov.
Article
in English
| MEDLINE | ID: mdl-32622142
18.
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.
Article
in English
| MEDLINE | ID: mdl-30880335
19.
Risk Factors and Outcomes of Nonmelanoma Skin Cancer in Children and Young Adults.
J Pediatr
; 211: 152-158, 2019 08.
Article
in English
| MEDLINE | ID: mdl-31103258
20.
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.
Article
in English
| MEDLINE | ID: mdl-31115156