Enzyme-Loaded Gel Core Nanostructured Lipid Carriers to Improve Treatment of Lysosomal Storage Diseases: Formulation and In Vitro Cellular Studies of Elosulfase Alfa-Loaded Systems

@article{lvarez2019EnzymeLoadedGC,
  title={Enzyme-Loaded Gel Core Nanostructured Lipid Carriers to Improve Treatment of Lysosomal Storage Diseases: Formulation and In Vitro Cellular Studies of Elosulfase Alfa-Loaded Systems},
  author={J. V. {\'A}lvarez and Carolina Herrero Filgueira and Alexandre de la Fuente Gonz{\'a}lez and Crist{\'o}bal Col{\'o}n Mejeras and A. Beiras Iglesias and S. Tomatsu and Jos{\'e} Blanco M{\'e}ndez and Asteria Luzardo {\'A}lvarez and M. Couce and F. O. Otero Espinar},
  journal={Pharmaceutics},
  year={2019},
  volume={11}
}
Mucopolysaccharidosis IVA (Morquio A) is a rare inherited metabolic disease caused by deficiency of the lysosomal enzyme N-acetylgalatosamine-6-sulfate-sulfatase (GALNS). Until now, treatments employed included hematopoietic stem cell transplantation and enzyme replacement therapy (ERT); the latter being the most commonly used to treat mucopolysaccharidoses, but with serious disadvantages due to rapid degradation and clearance. The purpose of this study was to develop and evaluate the potential… Expand
3 Citations
Enzyme replacement therapy for mucopolysaccharidoses; past, present, and future
Mucopolysaccharidosis IVA: Diagnosis, Treatment, and Management

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