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Sanofi Genzyme

SSIEM 2024 | Sep 3, 2024 - Sep 6, 2024

MPS1

Mucopolysaccharidosis Type I (MPS I) is one of seven inherited, multisystem, progressive disorders caused by a deficiency of the lysosomal enzyme α-L-iduronidase. Pathogenic variants of the IDUA gene lead to a defective enzyme with decreased activity of this enzyme causing lysosomal accumulation of GAGs, particularly dermatan and heparan sulfate. Over time, the build-up of GAGs leads to cellular dysfunction leading to cell death and organ-specific clinical manifestations.

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