Michael H. Gelb

Mass spectrometry measurements of glycosaminoglycans to assess disease-modifying treatments for the mucopolysaccharidoses: Methodological considerations.

The mucopolysaccharidoses are rare lysosomal disorders caused by defects in glycosaminoglycan (GAG) degradation, resulting in progressive multisystem disease. Biomarker measurement is central to assessing disease burden and therapeutic response, particularly as novel enzyme replacement, transplantation, gene and substr...