In editing this focused series on the novel advances in rare and undiagnosed liver diseases, we hoped to highlight, using specific examples, the burden of these diseases, their complexity, the unmet needs for the patients and their families, as well as the opportunities to learn more about pathophysiological mechanisms and targets that can also be applied to more common liver diseases.
Editorial
Rare and undiagnosed liver diseases: challenges and opportunities
Review Article
Novel approaches to liver disease diagnosis and modeling
Evolution of the liver biopsy and its future
Inherited iron overload disorders
Recent advances in Wilson disease
Alagille syndrome and non-syndromic paucity of the intrahepatic bile ducts
Alpha-1 antitrypsin deficiency liver disease
The acute hepatic porphyrias
Giant cell hepatitis associated with autoimmune hemolytic anemia: an update
Fibrocystic liver disease: novel concepts and translational perspectives
Hepatoblastoma: current knowledge and promises from preclinical studies
Liver transplantation for rare liver diseases and rare indications for liver transplant
Genome editing technologies to treat rare liver diseases
Undiagnosed liver diseases
Primary sclerosing cholangitis
Disclosure:
The series “Recent Advances in Rare Liver Diseases” was commissioned by the editorial office, Translational Gastroenterology and Hepatology without any sponsorship or funding. Luca Fabris and Mario Strazzabosco are serving as the unpaid Guest Editors for the series.