I am a Healthcare Professional

These posters, oral presentations, and/or abstracts are provided for medical and scientific purposes only, they may include investigational use or agents that are not approved by health authorities. The information presented is not meant to convey conclusions of safety or effectiveness prior to any regulatory approval from a health authority.

Sanofi does not recommend the use of any products in a manner inconsistent with that described in the full prescribing information. Please refer to the prescribing information in your country of practice for any medicinal products mentioned.

Availability of posters and/or abstracts on this site will conform with conference embargo policies. By selecting “Accept”, you are certifying that you are a Healthcare Professional.

Reject
Sanofi Genzyme

WORLDSymposium 2025 | Feb 3, 2025 - Feb 7, 2025

MPS I

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.

Poster