Detalhe da pesquisa
1.
Protocol paper: a multi-center, double-blinded, randomized, 6-month, placebo-controlled study followed by 12-month open label extension to evaluate the safety and efficacy of Saracatinib in Fibrodysplasia Ossificans Progressiva (STOPFOP).
BMC Musculoskelet Disord
; 23(1): 519, 2022 Jun 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-35650602
2.
Thrombocytosis in children and adolescents-classification, diagnostic approach, and clinical management.
Ann Hematol
; 100(7): 1647-1665, 2021 Jul.
Artigo
em Inglês
| MEDLINE | ID: mdl-33712866
3.
Bone marrow failure unresponsive to bone marrow transplant is caused by mutations in thrombopoietin.
Blood
; 130(7): 875-880, 2017 08 17.
Artigo
em Inglês
| MEDLINE | ID: mdl-28559357
4.
The thrombopoietin receptor P106L mutation functionally separates receptor signaling activity from thrombopoietin homeostasis.
Blood
; 125(7): 1159-69, 2015 Feb 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-25538044
5.
Novel Treatments for Rare Cancers: The U.S. Orphan Drug Act Is Delivering-A Cross-Sectional Analysis.
Oncologist
; 21(4): 487-93, 2016 Apr.
Artigo
em Inglês
| MEDLINE | ID: mdl-27022038
6.
First case of disseminated cryptococcosis in a Gorilla gorilla.
Mycoses
; 57(11): 664-71, 2014 Nov.
Artigo
em Inglês
| MEDLINE | ID: mdl-24963815
7.
Hereditary thrombocythemia caused by a thrombopoietin (THPO) gain-of-function mutation associated with multiple myeloma and congenital limb defects.
Ann Hematol
; 91(7): 1129-33, 2012 Jul.
Artigo
em Inglês
| MEDLINE | ID: mdl-22453305
8.
Fibrodysplasia Ossificans Progressiva: What Have We Achieved and Where Are We Now? Follow-up to the 2015 Lorentz Workshop.
Front Endocrinol (Lausanne)
; 12: 732728, 2021.
Artigo
em Inglês
| MEDLINE | ID: mdl-34858325
9.
13-cis retinoic acid treatment of a patient with chemotherapy refractory nephroblastomatosis.
J Pediatr Hematol Oncol
; 31(4): 296-9, 2009 Apr.
Artigo
em Inglês
| MEDLINE | ID: mdl-19346886
10.
Long-term remission of children with relapsed and secondary anaplastic large cell non-Hodgkin lymphoma (ALCL) following treatment with pulsed dexamethasone and low dose etoposide.
Pediatr Blood Cancer
; 50(1): 126-9, 2008 Jan.
Artigo
em Inglês
| MEDLINE | ID: mdl-16609950
11.
The uORF-containing thrombopoietin mRNA escapes nonsense-mediated decay (NMD).
Nucleic Acids Res
; 34(8): 2355-63, 2006.
Artigo
em Inglês
| MEDLINE | ID: mdl-16679454
12.
DNA methylation in PRDM8 is indicative for dyskeratosis congenita.
Oncotarget
; 7(10): 10765-72, 2016 Mar 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-26909595
13.
A novel autosomal recessive TERT T1129P mutation in a dyskeratosis congenita family leads to cellular senescence and loss of CD34+ hematopoietic stem cells not reversible by mTOR-inhibition.
Aging (Albany NY)
; 7(11): 911-27, 2015 Nov.
Artigo
em Inglês
| MEDLINE | ID: mdl-26546739
14.
Interaction of PSD-95 with potassium channels visualized by fluorescence lifetime-based resonance energy transfer imaging.
J Biomed Opt
; 9(4): 753-9, 2004.
Artigo
em Inglês
| MEDLINE | ID: mdl-15250762
15.
Pediatric case report on magnetic resonance imaging/transrectal ultrasound-fusion biopsy of rhabdomyosarcoma of the bladder/prostate: a new tool to reduce therapy-associated morbidity?
Urology
; 81(2): 417-20, 2013 Feb.
Artigo
em Inglês
| MEDLINE | ID: mdl-23374821
16.
Surface expression of inward rectifier potassium channels is controlled by selective Golgi export.
J Biol Chem
; 278(19): 17000-5, 2003 May 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-12609985