Work

Urinary glycosaminoglycan concentration as a biomarker for effectiveness of enzyme replacement and gene therapy in large animal models of mucopolysaccharidoses

Year: 2016

Type: article

Source: Molecular Genetics and Metabolism

DOI: 10.1016/j.ymgme.2015.12.214

Authors: Margret L. Casal, Ping Wang, Patricia A. ODonnell, Tracey Sikora, Therese Langan, Thomas O'Malley, Rita Ferla, Alberto Auricchio, James M. Wilson, Guangping Gao, Katherine Ponder, Mark E. Haskins

Institutions: University of Pennsylvania, Children's Hospital of Philadelphia, Telethon Institute Of Genetics And Medicine, University of Massachusetts Chan Medical School, Washington University in St. Louis

Language: English

Topic: Lysosomal Storage Disorders Research

Subfield: Physiology

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: closed

OpenAlex ID: W2266901718

FWCI:

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