Exercise no. 335 Odevixibat (KAYFANDA°) in Alagille syndrome

Alagille syndrome is a rare genetic disorder that affects a range of organs. Bile ducts are reduced in number, narrow and malformed, leading to cholestasis, usually from the first months of life, which results in an accumulation of bile acids in the liver and blood. Intense and persistent pruritus is a common clinical manifestation that profoundly impacts daily life, causing sleep disturbances, in particular. Other clinical manifestations include growth failure, bone fractures, cardiovascular disorders, intracranial haemorrhage, etc. About one-quarter of patients die before the age of 20 years, often due to cardiac or liver failure.