TIGEM is a multidisciplinary research Institute devoted to the study of the mechanisms underlying rare genetic diseases and the development of innovative therapies.

The Institute’s main goal is unite scientific knowledge, expertise, and education to focus efforts on: i) unique research at basic, pre-clinical, and translational levels that breaks new ground and defines scientific excellence; ii) new therapeutic approaches to advance the treatment of rare genetic diseases; iii) training the next generation of biomedical researchers; iv) the dissemination of TIGEM’s major discoveries and subsequent distribution to the wider public through partnerships with academic institutions and industry.
 

In the case of Wilson's disease, many challenges, issues and possible benefits for patients are identified : click here to see the presentation.