Extracellular matrix disruption is an early event in the pathogenesis of skeletal disease in mucopolysaccharidosis I
JM Heppner, F Zaucke, LA Clarke - Molecular genetics and metabolism, 2015 - Elsevier
Progressive skeletal and connective tissue disease represents a significant clinical burden
in all of the mucopolysaccharidoses. Despite the introduction of enzyme replacement
strategies for many of the mucopolysaccharidoses, symptomatology related to bone and
joint disease appears to be recalcitrant to current therapies. In order to address these unmet
medical needs a clearer understanding of skeletal and connective tissue disease
pathogenesis is required. Historically the pathogenesis of the mucopolysaccharidoses has …
in all of the mucopolysaccharidoses. Despite the introduction of enzyme replacement
strategies for many of the mucopolysaccharidoses, symptomatology related to bone and
joint disease appears to be recalcitrant to current therapies. In order to address these unmet
medical needs a clearer understanding of skeletal and connective tissue disease
pathogenesis is required. Historically the pathogenesis of the mucopolysaccharidoses has …
