Ontology highlight
ABSTRACT:
SUBMITTER: Alvarez JV
PROVIDER: S-EPMC6835858 | biostudies-literature | 2019 Oct
REPOSITORIES: biostudies-literature
Álvarez J Víctor JV Herrero Filgueira Carolina C González Alexandre de la Fuente AF Colón Mejeras Cristóbal C Beiras Iglesias Andrés A Tomatsu Shunji S Blanco Méndez José J Luzardo Álvarez Asteria A Couce María Luz ML Otero Espinar Francisco J FJ
Pharmaceutics 20191011 10
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 ...[more]