ERN RARE-LIVER

Gene transfer to the liver may be used to treat specific liver diseases, but can also induce hepatic protein production to treat conditions that do not affect the liver itself. While gene therapy is still in its infancy, preclinical and clinical trials have already shown promising results in several diseases. For instance, in Hemophilia A, an X-linked bleeding disorder due to deficiencies in coagulation factor VIII, multiple clinical trials with AAV-vector liver-directed gene therapy have reported promising results in restoring therapeutically relevant FVIII levels with sustained production for years (1).  [read more]

There are insufficient population-level data on the effects of primary sclerosing cholangitis (PSC) in patients with inflammatory bowel disease (IBD).  [read more]

Cholangiocarcinoma (CCA) includes a cluster of highly heterogeneous biliary malignant tumours that can arise at any point of the biliary tree. Their incidence is increasing globally, currently accounting for 15% of all primary liver cancers and 3% of gastrointestinal malignancies. [read more]