Search details
1.
Gene therapy restores the transcriptional program of hematopoietic stem cells in Fanconi anemia.
Haematologica
; 108(10): 2652-2663, 2023 10 01.
Article
in English
| MEDLINE | ID: mdl-37021532
2.
Natural gene therapy by reverse mosaicism leads to improved hematology in Fanconi anemia patients.
Am J Hematol
; 96(8): 989-999, 2021 08 01.
Article
in English
| MEDLINE | ID: mdl-33984160
3.
Mitapivat versus Placebo for Pyruvate Kinase Deficiency.
N Engl J Med
; 386(26): 2538-2539, 2022 06 30.
Article
in English
| MEDLINE | ID: mdl-35767453
4.
Mosaicism in Fanconi anemia: concise review and evaluation of published cases with focus on clinical course of blood count normalization.
Ann Hematol
; 99(5): 913-924, 2020 May.
Article
in English
| MEDLINE | ID: mdl-32065290
5.
Minimal conditioning in Fanconi anemia promotes multi-lineage marrow engraftment at 10-fold lower cell doses.
J Gene Med
; 20(10-11): e3050, 2018 10.
Article
in English
| MEDLINE | ID: mdl-30129972
6.
Tisagenlecleucel in Diffuse Large B-Cell Lymphoma.
N Engl J Med
; 380(16): 1585-1586, 2019 04 18.
Article
in English
| MEDLINE | ID: mdl-30995383
7.
Who should be eligible for gene therapy clinical trials in red blood cell pyruvate kinase deficiency (PKD)?: Toward an expanded definition of severe PKD.
Am J Hematol
; 97(3): E120-E125, 2022 03 01.
Article
in English
| MEDLINE | ID: mdl-34989415
8.
Ramucirumab versus placebo as second-line treatment in patients with advanced hepatocellular carcinoma following first-line therapy with sorafenib (REACH): a randomised, double-blind, multicentre, phase 3 trial.
Lancet Oncol
; 16(7): 859-70, 2015 Jul.
Article
in English
| MEDLINE | ID: mdl-26095784
9.
Ramucirumab monotherapy for previously treated advanced gastric or gastro-oesophageal junction adenocarcinoma (REGARD): an international, randomised, multicentre, placebo-controlled, phase 3 trial.
Lancet
; 383(9911): 31-39, 2014 Jan 04.
Article
in English
| MEDLINE | ID: mdl-24094768
10.
Ramucirumab plus paclitaxel versus placebo plus paclitaxel in patients with previously treated advanced gastric or gastro-oesophageal junction adenocarcinoma (RAINBOW): a double-blind, randomised phase 3 trial.
Lancet Oncol
; 15(11): 1224-35, 2014 Oct.
Article
in English
| MEDLINE | ID: mdl-25240821
11.
A phase 2, single-arm study of ramucirumab in patients with metastatic renal cell carcinoma with disease progression on or intolerance to tyrosine kinase inhibitor therapy.
Cancer
; 120(11): 1647-55, 2014 Jun 01.
Article
in English
| MEDLINE | ID: mdl-24577874
12.
An open-label phase II study evaluating the safety and efficacy of ramucirumab combined with mFOLFOX-6 as first-line therapy for metastatic colorectal cancer.
Oncologist
; 19(4): 350-1, 2014 Apr.
Article
in English
| MEDLINE | ID: mdl-24674871
13.
A phase II study of ramucirumab (IMC-1121B) in the treatment of persistent or recurrent epithelial ovarian, fallopian tube or primary peritoneal carcinoma.
Gynecol Oncol
; 134(3): 478-85, 2014 Sep.
Article
in English
| MEDLINE | ID: mdl-25016924
14.
Thrombotic microangiopathy following systemic AAV administration is dependent on anti-capsid antibodies.
J Clin Invest
; 134(1)2024 Jan 02.
Article
in English
| MEDLINE | ID: mdl-37988172
15.
Advances in NK cell therapy for brain tumors.
NPJ Precis Oncol
; 7(1): 17, 2023 Feb 15.
Article
in English
| MEDLINE | ID: mdl-36792722
16.
Predicting disease progression and the need for tumor-directed treatment in tectal plate gliomas.
J Neurosurg Pediatr
; 32(3): 332-342, 2023 Sep 01.
Article
in English
| MEDLINE | ID: mdl-37347621
17.
Copeptin levels in hospitalized infants and children with suspected vasopressin-dependent disorders: a case series.
J Pediatr Endocrinol Metab
; 36(5): 492-499, 2023 May 25.
Article
in English
| MEDLINE | ID: mdl-37029788
18.
Drug delivery across the blood-brain barrier for the treatment of pediatric brain tumors - An update.
Adv Drug Deliv Rev
; 185: 114303, 2022 06.
Article
in English
| MEDLINE | ID: mdl-35460714
19.
Findings on Optical Coherence Tomography in Malignant Infantile Osteopetrosis.
Ophthalmic Surg Lasers Imaging Retina
; 53(7): 398-402, 2022 07.
Article
in English
| MEDLINE | ID: mdl-35858232
20.
Preclinical safety and efficacy of lentiviral-mediated gene therapy for leukocyte adhesion deficiency type I.
Mol Ther Methods Clin Dev
; 26: 459-470, 2022 Sep 08.
Article
in English
| MEDLINE | ID: mdl-36092365