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TRANSLATIONAL RESEARCH

What is the link between
WilsonMed and copper?

Wilson's disease (WD) is a rare inherited condition that causes copper levels to build up in several organs, especially the liver, brain and eyes. WilsonMed is a collaborative approach to research, management and treatment of patients with WD involving scientists, clinicians, industry and patients.

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New therapeutic approaches for Wilson’s disease

WilsonMed is a significant breakthrough in the management and treatment of patients with Wilson's disease through the synergistic combination of scientific expertise and patient experience.

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Improving current therapies

Wilson disease (WD) is a genetic rare disease that results from a defect of copper (Cu) transporter ATP7B leading to progressive Cu accumulation. Currently, therapy is based on anti-Cu compounds with lifelong prescriptions.

Due to limitations of current therapy, improved therapy has to be developed to counteract devastating conditions of patients and their families.

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Patient involvement in preclinical development

WilsonMed aims to complete the preclinical development of selected anti-Cu compounds. The validated anti-Cu therapies will be developed for the market by all partners through the post-project steps of financing, formulation development, toxicology, clinical trials and regulatory approval.

The involvement of patient organisations throughout the project is crucial.

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Increasing knowledge on the mode of action

The new therapeutic approach includes advanced drug delivery into the brain and liver and developing animal models with further refinement

WilsonMed will thereby increase knowledge of the mode of action (MoA) of novel anti-Cu compounds as required by regulatory authorities and will protect th e knowledge generated to support the exploitation/commercialization of the results.

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WilsonMed is a unique partnership that brings together leading experts in basic/translational research, clinicians, and Wilson disease patient organisations to develop new treatment options, initiate proof-of-principle studies in preclinical models, evaluate predictive biomarkers for monitoring therapy, and bring new concepts to patients first-hand. The WilsonMed project has received funding from the EJP RD initiative of the European Union’s Horizon 2020 research and innovation programme.

Researchers

logo researcher Hartmut SCHMIDT
Hartmut SCHMIDT

PRINCIPAL INVESTIGATOR AND CONSORTIUM COORDINATOR

Direktor der Klinik für Gastroenterologie, Hepatologie und Transplantationsmedizin Universitätsklinikum Essen Universität Duisburg-Essen
E-mail: gastroenterologie(at)uk-essen.de
Germany

logo researcher Guido KROEMER
Guido KROEMER

PRINCIPAL INVESTIGATOR

Director of Research Inserm U1138 "Metabolism, cancer and immunity". Institut National de la Santé et la Recherche Médicale (Inserm)
France

logo researcher Roman POLISHCHUK
Roman POLISHCHUK

PRINCIPAL INVESTIGATOR

Associate Investigator Head of Advanced Microscopy and Imaging Core Telethon Institute of Genetics and Medicine (Tigem)
Italy

logo researcher Piotr SOCHA
Piotr SOCHA

PRINCIPAL INVESTIGATOR AND CLINICAL EXPERT

Professor in the Department of Gastroenterology, Hepatology, Nutritional Disorders and Pediatrics. Deputy Director for Research The Children’s Memorial Health Insitute (CMHI)
Poland.

logo researcher Hans ZISCHKA
Hans ZISCHKA

PRINCIPAL INVESTIGATOR

Group Leader “Oxidative Cell Death” Institute of Molecular Toxicology and Pharmacology, Helmholtz Center Munich, Germany.
Research Center for Environmental Health,
Germany

Patient advocacy organisations (PAO)

logo patient Wiebke PAPENTHIN
Wiebke PAPENTHIN

PATIENT REPRESENTATIVE

Morbus Wilson e.V.
Germany

logo patient Salvatore Di LORENZO
Salvatore Di LORENZO

PATIENT REPRESENTATIVE

Associazione Nazionale Malattia di Wilson
Italy

logo patient Tomasz LITWIN
Tomasz LITWIN

PATIENT REPRESENTATIVE

Polish patients group collaborating with Dr SOCHA
Poland

logo patient Caroline ROATTA
Caroline ROATTA

PATIENT REPRESENTATIVE AND PAO COORDINATOR

French patients group. Ms Roatta is also the project's patient coordinator.
France


News and Events

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2024-08-22

New project publications 2024

The WilsonMed scientific team published two papers this summer. They are available in the ‘Resources’ section.

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2024-05-10

Highlighting the work of polish support group 'Living with Wilson's Disease'

Per Humanus foundation presents the key points, needs and the actions implemented to support patients during the WilsonMed Project.

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2024-05-07

International Wilson's disease patient collaboration

Representatives of european and american patient organisations met for the second time at Aarhus

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