ERN RARE-LIVER

The manuscript on COVID-19 in patients with autoimmune liver diseases, based on data from the R-LIVER COVID-19 registry and in cooperation with the EASL and international COVID-19 registry, was recently accepted for publication by the Journal of Hepatology. [read more]

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]