The Telethon Institute of Genetics and Medicine (TIGEM), led by Andrea Ballabio, has the sole purpose of executing top quality research to understand the mechanisms behind rare genetic diseases, and furthermore, to apply new knowledge to the development of novel treatments. Overlooked by the pharmaceutical industry, rare genetic diseases are hardly met with the attention that they deserve.

TIGEM is devoted to making these disorders its top priority and uses interdisciplinary techniques to make the most complete effort possible. TIGEM's research staff and students organize projects which are funded by Telethon, the European Union, and various institutions. The Institute's talented members, in combination with funding from important institutions, have made TIGEM successful in making key biological discoveries.