Designing Clinical Trials in Wilson’s Disease,Hepatology

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Designing Clinical Trials in Wilson’s Disease
Hepatology ( IF 12.9 ) Pub Date : 2021-07-28 , DOI: 10.1002/hep.32074
Peter Ott ₁ , Aftab Ala _{2,

3,

4} , Frederick K Askari ₅ , Anna Czlonkowska ₆ , Ralf-Dieter Hilgers ₇ , Aurélia Poujois ₈ , Eve A Roberts ₉ , Thomas Damgaard Sandahl ₁ , Karl Heinz Weiss _{10,

11} , Peter Ferenci ₁₂ , Michael L Schilsky ₁₃

Affiliation

Department of Hepatology and Gastroenterology, Aarhus University, Aarhus, Denmark.
Department of Gastroenterology and Hepatology, The Royal Surrey NHS Foundation Trust, Guildford, United Kingdom.
Department of Clinical and Experimental Medicine, University of Surrey, Guildford, United Kingdom.
Institute of Liver Studies, King's College Hospital, London, United Kingdom.
Department of Medicine, University of Michigan, Ann Arbor, MI, USA.
Second Department of Neurology, Institute of Psychiatry and Neurology, Warsaw, Poland.
Department of Medical Statistics, RWTH Aachen University, Aachen, Germany.
Neurology Department and National Reference Centre for Wilson's Disease, Rothschild Foundation Hospital, Paris, France.
Departments of Paediatrics, Medicine, and Pharmacology and Toxicology, University of Toronto, Toronto, Ontario, Canada.
Salem Medical Center, Heidelberg, Germany.
Department of Internal Medicine, IV at University Hospital Heidelberg, Heidelberg, Germany.
Department of Gastroenterology and Hepatology, Medical University of Vienna, Vienna, Austria.
Departments of Medicine and Surgery, Yale University Medical Center, New Haven, CT, USA.

Wilson’s disease (WD) is an autosomal-recessive disorder caused by ATP7B gene mutations leading to pathological accumulation of copper in the liver and brain. Adoption of initial treatments for WD was based on empirical observations. These therapies are effective, but there are still unmet needs for which treatment modalities are being developed. An increase of therapeutical trials is anticipated.

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