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

MDS 2024 | Sep 27, 2024 - Oct 1, 2024

Gaucher

Gaucher disease is an autosomal recessive disorder caused by a deficiency in glucocerebrosidase (GBA) enzyme activity. Deficiency or absence of the enzyme leads to buildup of glycosylceramide (GL-1) and glucosylsphingosine (lyso-GL-1). Three clinical types are delineated by the absence (type 1) or presence (types 2 and 3) of primary CNS involvement. Type 3 disease is a chronic neuronopathic disorder with wide-ranging effects and diverse neurological manifestations.

Poster