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1.
Enzyme replacement therapy with velmanase alfa (human recombinant alpha-mannosidase): Novel global treatment response model and outcomes in patients with alpha-mannosidosis.
Mol Genet Metab
; 124(2): 152-160, 2018 06.
Artículo
en Inglés
| MEDLINE | ID: mdl-29716835
2.
Efficacy and safety of Velmanase alfa in the treatment of patients with alpha-mannosidosis: results from the core and extension phase analysis of a phase III multicentre, double-blind, randomised, placebo-controlled trial.
J Inherit Metab Dis
; 41(6): 1215-1223, 2018 11.
Artículo
en Inglés
| MEDLINE | ID: mdl-29846843
3.
Comprehensive long-term efficacy and safety of recombinant human alpha-mannosidase (velmanase alfa) treatment in patients with alpha-mannosidosis.
J Inherit Metab Dis
; 41(6): 1225-1233, 2018 11.
Artículo
en Inglés
| MEDLINE | ID: mdl-29725868
4.
In vivo functional and transcriptional profiling of bone marrow stem cells after transplantation into ischemic myocardium.
Arterioscler Thromb Vasc Biol
; 32(1): 92-102, 2012 Jan.
Artículo
en Inglés
| MEDLINE | ID: mdl-22034515
5.
How to START? Four pillars to optimally begin your orphan drug development.
Orphanet J Rare Dis
; 18(1): 229, 2023 08 03.
Artículo
en Inglés
| MEDLINE | ID: mdl-37537670
6.
Enabling heterogeneous data integration and biomedical event prediction through ICT: the test case of cancer reoccurrence.
Adv Exp Med Biol
; 696: 367-75, 2011.
Artículo
en Inglés
| MEDLINE | ID: mdl-21431577
7.
Use of the Bruininks-Oseretsky test of motor proficiency (BOT-2) to assess efficacy of velmanase alfa as enzyme therapy for alpha-mannosidosis.
Mol Genet Metab Rep
; 23: 100586, 2020 Jun.
Artículo
en Inglés
| MEDLINE | ID: mdl-32292699
8.
Clinically driven semi-supervised class discovery in gene expression data.
Bioinformatics
; 24(16): i90-7, 2008 Aug 15.
Artículo
en Inglés
| MEDLINE | ID: mdl-18689846
9.
The use or generation of biomedical data and existing medicines to discover and establish new treatments for patients with rare diseases - recommendations of the IRDiRC Data Mining and Repurposing Task Force.
Orphanet J Rare Dis
; 14(1): 225, 2019 10 15.
Artículo
en Inglés
| MEDLINE | ID: mdl-31615551
10.
Correction: Identification of an early transcriptomic signature of insulin resistance and related diseases in lymphomonocytes of healthy subjects.
PLoS One
; 14(1): e0211394, 2019.
Artículo
en Inglés
| MEDLINE | ID: mdl-30673781
11.
Addressing challenges in the diagnosis and treatment of rare genetic diseases.
Nat Rev Drug Discov
; 17(3): 151-152, 2018 03.
Artículo
en Inglés
| MEDLINE | ID: mdl-29242613
12.
Navigating Market Authorization: The Path Holoclar Took to Become the First Stem Cell Product Approved in the European Union.
Stem Cells Transl Med
; 7(1): 146-154, 2018 01.
Artículo
en Inglés
| MEDLINE | ID: mdl-29280318
13.
Circulating chemokines accurately identify individuals with clinically significant atherosclerotic heart disease.
Physiol Genomics
; 31(3): 402-9, 2007 Nov 14.
Artículo
en Inglés
| MEDLINE | ID: mdl-17698927
14.
Identification of an early transcriptomic signature of insulin resistance and related diseases in lymphomonocytes of healthy subjects.
PLoS One
; 12(8): e0182559, 2017.
Artículo
en Inglés
| MEDLINE | ID: mdl-28777829
15.
Dietary glycemic index and liver steatosis.
Am J Clin Nutr
; 84(1): 136-42; quiz 268-9, 2006 Jul.
Artículo
en Inglés
| MEDLINE | ID: mdl-16825687
16.
Relation of plasma insulin levels to forearm flow-mediated dilatation in healthy volunteers.
Am J Cardiol
; 97(8): 1250-4, 2006 Apr 15.
Artículo
en Inglés
| MEDLINE | ID: mdl-16616036
17.
Long-Term Retrospective Analysis of Gene Therapy with Alipogene Tiparvovec and Its Effect on Lipoprotein Lipase Deficiency-Induced Pancreatitis.
Hum Gene Ther
; 27(11): 916-925, 2016 11.
Artículo
en Inglés
| MEDLINE | ID: mdl-27412455
18.
Relationship between leptin, insulin, body composition and liver steatosis in non-diabetic moderate drinkers with normal transaminase levels.
Eur J Endocrinol
; 153(2): 283-90, 2005 Aug.
Artículo
en Inglés
| MEDLINE | ID: mdl-16061835
19.
Hyperinsulinemia predicts hepatic fat content in healthy individuals with normal transaminase concentrations.
Metabolism
; 54(12): 1566-70, 2005 Dec.
Artículo
en Inglés
| MEDLINE | ID: mdl-16311087
20.
Boosting delivery of rare disease therapies: the IRDiRC Orphan Drug Development Guidebook.
Nat Rev Drug Discov
; 19(8): 495-496, 2020 08.
Artículo
en Inglés
| MEDLINE | ID: mdl-32313251