What is it about?
This poster describes the groups work to develop an enzyme replacement for Sanfilippo IIID (MPS IIID).
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Why is it important?
There is no treatment for this disease. An ERT would be the first treatment developed.
Perspectives
This work has been funded by an NINDS STTR. This poster describes the initial work from a phase I grant.
Dr Sean Ekins
Collaborations in Chemistry
Read the Original
This page is a summary of: Uptake and lysosomal delivery of recombinant human alpha-n-acetylglucosamine-6-sulfatase to mucopolysaccharidosis IIID fibroblasts, Molecular Genetics and Metabolism, February 2016, Elsevier,
DOI: 10.1016/j.ymgme.2015.12.365.
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