Evaluation of Non-Reducing End Pathologic Glycosaminoglycan Detection Method for Monitoring Therapeutic Response to Enzyme Replacement Therapy in Human Mucopolysaccharidosis I

Therapeutic development and monitoring require demonstration of effects on disease phenotype. However, due to the complexity of measuring clinically-relevant effects in rare multisystem diseases, robust biomarkers are essential. For the mucopolysaccharidoses (MPS), the measurement of glycosaminoglyc...

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Publicat a:Mol Genet Metab
Autors principals: Vera, Moin U., Le, Steven Q., Victoroff, Alla, Passage, Merry B., Brown, Jillian, Crawford, Brett E., Polgreen, Lynda E., Chen, Agnes H., Dickson, Patricia I.
Format: Artigo
Idioma:Inglês
Publicat: 2019
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Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC7219480/
https://ncbi.nlm.nih.gov/pubmed/31630958
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgme.2019.09.001
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