α-galactosidase A - Resultats de la cerca - Dipòsit Digital de Documents de la UAB

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Resultats globals: 2 registres trobats en 0.03 segons.
Articles, 2 registres trobats

Articles	2 registres trobats

9 p, 745.9 KB

Long-term follow-up of renal function in patients treated with migalastat for Fabry disease / Bichet, Daniel G. (Department of Medicine, Hôpital du Sacré-Coeur, University of Montréal, Montreal, Quebec, Canada) ; Torra Balcells, Roser (Institut d'Investigació Biomèdica Sant Pau) ; Wallace, Eric (Department of Medicine, University of Alabama, Birmingham, AL, USA) ; Hughes, Derralynn A (Lysosomal Storage Disorders Unit, Royal Free London NHS Foundation Trust and University College London, London, UK) ; Giugliani, Roberto (Instituto Nacional de Genética Médica Populacional (Porto Alegre, Brasil)) ; Skuban, Nina (Amicus Therapeutics, Inc., Cranbury, NJ, USA) ; Krusinska, Eva (Amicus Therapeutics, Inc., Cranbury, NJ, USA) ; Feldt-Rasmussen, Ulla (Copenhagen University Hospital Rigshospitalet) ; Schiffmann, Raphael (Institute of Metabolic Disease, Baylor Scott & White Research Institute, Dallas, TX, USA) ; Nicholls, Kathy (Royal Melbourne Hospital (Melbourne, Austràlia)) ; Universitat Autònoma de Barcelona
The effect of migalastat on long-term renal outcomes in enzyme replacement therapy (ERT)-naive and ERT-experienced patients with Fabry disease is not well defined. An integrated posthoc analysis of the phase 3 clinical trials and open-label extension studies was conducted to evaluate long-term changes in renal function in patients with Fabry disease and amenable GLA variants who were treated with migalastat for ≥2 years during these studies. [...]
2021 - DOI (Digital Object Identifier)

10.1016/j.ymgmr.2021.100786
Molecular Genetics and Metabolism Reports, Vol. 28 (august 2021)

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47 p, 816.8 KB

Highly versatile polyelectrolyte complexes for improving the enzyme replacement therapy of lysosomal storage disorders / Giannotti, M. I. (Universitat de Barcelona. Departament de Química Física) ; Abasolo, Ibane (Hospital Universitari Vall d'Hebron. Institut de Recerca) ; Oliva, Mireia (Universitat de Barcelona. Departament de Farmàcia i Tecnologia Farmacèutica, i Fisicoquímica) ; Andrade, Fernanda (Universitat de Barcelona. Departament de Farmàcia i Tecnologia Farmacèutica, i Fisicoquímica) ; García Aranda, Natalia (Hospital Universitari Vall d'Hebron. Institut de Recerca) ; Melgarejo, Marta (Parc Científic de Barcelona) ; Pulido, Daniel (Parc Científic de Barcelona) ; Corchero Nieto, José Luis (Universitat Autònoma de Barcelona. Departament de Genètica i de Microbiologia) ; Fernández Caparrós, Yolanda (Hospital Universitari Vall d'Hebron. Institut de Recerca) ; Villaverde Corrales, Antonio (Universitat Autònoma de Barcelona. Institut de Biotecnologia i de Biomedicina "Vicent Villar Palasí") ; Royo, Miriam (Parc Científic de Barcelona) ; García Parajó, María F.. (Institut de Ciències Fotòniques) ; Sanz, Fausto (Parc Científic de Barcelona) ; Schwartz, Simó Jr (Hospital Universitari Vall d'Hebron. Institut de Recerca)
Lysosomal storage disorders are currently treated by enzyme replacement therapy (ERT) through the direct administration of the unprotected recombinant protein to the patients. Herein we present an ionically crosslinked polyelectrolyte complex (PEC) composed of trimethyl chitosan (TMC) and -galactosidase A (GLA), the defective enzyme in Fabry disease, with the capability of directly targeting endothelial cells by incorporating peptide ligands containing the RGD sequence. [...]
2016 - DOI (Digital Object Identifier)

10.1021/acsami.6b08356
ACS applied materials & interfaces, Vol. 8 Núm 39 (Octubre 2016) , p. 25741-25752

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