UAB Digital Repository of Documents 5 records found  Search took 0.02 seconds. 
1.
5 p, 250.5 KB Elosulfase alfa for mucopolysaccharidosis type IVA : Real-world experience in 7 patients from the Spanish Morquio-A early access program / Pintos Morell, Guillem (Institut Germans Trias i Pujol. Hospital Universitari Germans Trias i Pujol) ; Blasco Alonso, Javier (Hospital Regional Universitario de Málaga) ; Couce, María L. (Hospital Clínico Universitario de Santiago) ; Gutiérrez-Solana, Luís G. (Hospital Infantil Universitario Niño Jesús) ; Guillén-Navarro, Encarna (IMIB - Arrixaca, Murcia) ; O'Callaghan, Mar (Hospital Sant Joan de Déu, Esplugues, Barcelona) ; del Toro, Mireia (Hospital Universitari Vall d'Hebron) ; Universitat Autònoma de Barcelona
There is a growing interest in evaluating the effectiveness of enzyme replacement therapy (ERT) with elosulfase alfa in patients with mucopolysaccharidosis type IVA (MPS-IVA) under real-world conditions. [...]
2018 - 10.1016/j.ymgmr.2018.03.009
Molecular Genetics and Metabolism Reports, Vol. 15 (april 2018) , p. 116-120  
2.
15 p, 4.6 MB Hepatic regulation of VLDL receptor by PPARβ/δ and FGF21 modulates non-alcoholic fatty liver disease / Zarei, Mohammad (Universitat de Barcelona. Departament de Farmacologia, Toxicologia i Química Terapèutica) ; Barroso, Emma (Universitat de Barcelona. Departament de Farmacologia, Toxicologia i Química Terapèutica) ; Palomer, Xavier (Universitat de Barcelona. Departament de Farmacologia, Toxicologia i Química Terapèutica) ; Dai, Jianli (University of the Chinese Academy of Sciences. Institute for Nutritional Sciences. Key Laboratory of Nutrition and Metabolism,) ; Rada, Patricia (Instituto de Salud Carlos III. Centro de Investigación Biomédica en Red) ; Quesada-López, Tania (Universitat de Barcelona. Departament de Bioquímica i Biomedicina Molecular) ; Escolà-Gil, Joan Carles (Universitat Autònoma de Barcelona. Departament de Bioquímica i Biología Molecular) ; Cedó, Lidia (Institut d'Investigació Biomèdica Sant Pau) ; Zali, Mohammad Reza (Shahid Beheshti University of Medical Sciences. Research Institute for Gastroenterology and Liver Diseases) ; Molaei, Mahsa (Shahid Beheshti University of Medical Sciences. Research Institute for Gastroenterology and Liver Diseases) ; Dabiri, Reza (Semnan University of Medical Sciences. lnternal Medicine Department) ; Vázquez Cruz, Santiago (Universitat de Barcelona. Departament de Farmacologia, Toxicologia i Química Terapèutica) ; Pujol, Eugènia (Universitat de Barcelona. Departament de Farmacologia, Toxicologia i Química Terapèutica) ; Valverde, Ángela M. (Instituto de Investigaciones Biomédicas Alberto Sols) ; Villarroya, Francesc (Universitat de Barcelona. Departament de Bioquímica i Biomedicina Molecular) ; Liu, Yong (Wuhan University. Institute for Advanced Studies) ; Wahli, Walter (Nanyang Technological University. Lee Kong Chian School of Medicine) ; Vázquez-Carrera, Manuel (Universitat de Barcelona. Departament de Farmacologia, Toxicologia i Química Terapèutica) ; Universitat Autònoma de Barcelona
The very low-density lipoprotein receptor (VLDLR) plays an important role in the development of hepatic steatosis. In this study, we investigated the role of Peroxisome Proliferator-Activated Receptor (PPAR)β/δ and fibroblast growth factor 21 (FGF21) in hepatic VLDLR regulation. [...]
2018 - 10.1016/j.molmet.2017.12.008
Molecular metabolism, Vol. 8 (Feb. 2018) , p. 117-131  
3.
5 p, 246.4 KB Long-term outcomes with agalsidase alfa enzyme replacement therapy : Analysis using deconstructed composite events / Beck, Michael (University Medical Center, Mainz, Germany) ; Hughes, Derralynn (University College London) ; Kampmann, Christoph (University Medical Center, Mainz, Germany) ; Pintos Morell, Guillem (Institut Germans Trias i Pujol) ; Ramaswami, Uma (University College London) ; West, Michael L. (Dalhousie University, Halifax, Canada) ; Giugliani, Roberto (Universidade Federal do Rio Grande do Sul, Brazil) ; Universitat Autònoma de Barcelona
This is a retrospective analysis of Fabry Outcome Survey data from children/adults (n = 677) receiving agalsidase alfa enzyme replacement therapy for a median of 3 years, examining cerebrovascular, cardiac, and renal morbidity endpoints separately. [...]
2017 - 10.1016/j.ymgmr.2017.10.008
Molecular Genetics and Metabolism Reports, Vol. 14 (november 2017) , p. 31-35  
4.
17 p, 5.4 MB AAV-mediated pancreatic overexpression of Igf1 counteracts progression to autoimmune diabetes in mice / Mallol, Cristina (CIBER de Diabetes y Enfermedades Metabólicas Asociadas) ; Casaña Lorente, Estefania (Universitat Autònoma de Barcelona. Centre de Biotecnologia Animal i de Teràpia Gènica) ; Jimenez, Veronica (CIBER de Diabetes y Enfermedades Metabólicas Asociadas) ; Casellas, Alba (CIBER de Diabetes y Enfermedades Metabólicas Asociadas) ; Haurigot Mendonça, Virginia (CIBER de Diabetes y Enfermedades Metabólicas Asociadas) ; Jambrina, Claudia (Universitat Autònoma de Barcelona. Centre de Biotecnologia Animal i de Teràpia Gènica) ; Sacristan, Victor (Universitat Autònoma de Barcelona. Centre de Biotecnologia Animal i de Teràpia Gènica) ; Morró Larrubia, Meritxell (CIBER de Diabetes y Enfermedades Metabólicas Asociadas) ; Agudo, Judith (CIBER de Diabetes y Enfermedades Metabólicas Asociadas) ; Vilà, Laia (CIBER de Diabetes y Enfermedades Metabólicas Asociadas) ; Bosch i Tubert, Fàtima (CIBER de Diabetes y Enfermedades Metabólicas Asociadas)
Type 1 diabetes is characterized by autoimmune destruction of β-cells leading to severe insulin deficiency. Although many improvements have been made in recent years, exogenous insulin therapy is still imperfect; new therapeutic approaches, focusing on preserving/expanding β-cell mass and/or blocking the autoimmune process that destroys islets, should be developed. [...]
2017 - 10.1016/j.molmet.2017.05.007
Molecular metabolism, Vol. 6 (may 2017) , p. 664-680  
5.
7 p, 412.7 KB Long-term effectiveness of agalsidase alfa enzyme replacement in Fabry disease : A Fabry Outcome Survey analysis / Beck, Michael (University Medical Center, University of Mainz, Department of Paediatrics) ; Hughes, Derralynn (Royal Free London NHS Foundation Trust, University College of London) ; Kampmann, Christoph (University Medical Center, University of Mainz, Department of Paediatrics) ; Larroque, Sylvain (Shire, Zug) ; Mehta, Atul (Royal Free London NHS Foundation Trust, University College of London) ; Pintos-Morellell, Guillem (Institut Germans Trias i Pujol. Hospital Universitari Germans Trias i Pujol. Departament de Pediatria) ; Ramaswami, Uma (Royal Free London NHS Foundation Trust, University College of London) ; West, Michael (Department of Medicine, Dalhousie University) ; Wijatyk, Anna (Shire) ; Giugliani, Roberto (Medical Genetics Service HCPA/Dep Genet UFRGS and INAGEMP, Porto Alegre) ; Universitat Autònoma de Barcelona
Outcomes from 5 years of treatment with agalsidase alfa enzyme replacement therapy (ERT) for Fabry disease in patients enrolled in the Fabry Outcome Survey (FOS) were compared with published findings for untreated patients with Fabry disease. [...]
2015 - 10.1016/j.ymgmr.2015.02.002
Molecular Genetics and Metabolism Reports, Vol. 3 (march 2015) , p. 21-27  

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