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Sanofi Genzyme

WORLDSymposium 2022 | Feb 7, 2022 - Feb 11, 2022

MPS I

Mucopolysaccharidosis Type I (MPS I) is one of the seven mucopolysaccharidoses. It is an inherited, multisystem, progressive disorder caused by a deficiency of the lysosomal enzyme α-L-iduronidase, which is encoded by the IDUA gene. Pathogenic mutations to the IDUA gene lead to a defective enzyme with lesser than normal alpha-L-iduronidase activity. The decreased activity of this enzyme causes GAGs, particularly dermatan and heparan sulfate, to accumulate in the lysosome. Over time the build-up of dermatan and heparan sulfate leads to excess cellular storage and cellular dysfunction which ultimately leads to cell death and organ-specific clinical manifestations. The tissues and organs which are affected varies.

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