In this ERN Rare-Liver webinar, held on 12 December 2023, Prof. Strnad and Dr Ruiz gave an exciting update to the alpha-1 antitrypsin deficiency. The presentation covered “siRNA therapy in hepatology: a1-AT deficiency”.

Targeted Audience

  • General practitioners
  • Hepatologists
  • Oncologists
  • Health care professionals

Alpha-1 antitrypsin deficiency: A poster child for nucleic acid therapy


Pavel Strnad Pavel Strnad is a full professor and leading physician at the University Hospital Aachen, Germany. Since 2008, he is heading his own research lab focusing on translational gastroenterology. In 2016, he was named one of the Rising Stars of the United European Gastroenterology. He is heading the European initiative for study of alpha1-antitrypsin deficiency-associated liver disease. His interest include (but are not limited to) metabolic liver disease, liver cirrhosis and complications, keratins and iron metabolism. His clinical focus is on emerging drugs, end-stage liver disease and liver transplantation.
Mathias Ruiz Mathias Ruiz is a paediatric hepatologist, gastroenterologist and nutritionist at the Hospital Femme-Mère-Enfant in Lyon, the second-largest university hospital in France, a reference centre for biliary atresia and genetic cholestastic diseases, and one of the paediatric liver transplantation centres in France. He is a member of the European Reference Network (ERN) Rare-Liver, co-leading the paediatric part of the alpha1-antitrypsin deficiency working-group, and he is a member of the Hepatology Interest Group of the European Society of Paediatric Gastroenterology Hepatology and Nutrition (ESPGHAN). His research mainly focuses on paediatric liver diseases, in particular alpha1-antitrypsin deficiency, biliary atresia and genetic cholestasis, as well as paediatric liver transplantation.
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