what we fight

 

Among the more than 500 types of hereditary metabolic diseases known today, there is a group of about 40 diseases, called lysosomal storage disorders, manifested by significant irreversible degenerative effects against brain and central nervous system.

In these cases the damage is mainly due to an accumulation of substances in cells, which, due to genetic alteration, can no longer perform their function of "chemical laboratory" and break down what they normally transform into energy. In this way the body is intoxicated and will soon interrupt the biological functions essential to life.

 

Are Fighting with us

The Telethon Institute of Genetics and Medicine (Tigem), directed by Andrea Ballabio, created in 1994 to promote research aimed at the diagnosis, prevention and treatment of human genetic diseases in collaboration with the ’University of Naples.

 

 

 

The "Mario Negri" Institute for Pharmacological Research and the San Raffaele-Telethon Institute for Gene Therapy of Genetic Disease (HSR-TIGET) whose mission is the clinical application of gene therapy, and particularly Alessandra Biffi   coordinator of the research project aimed at investigating the possible therapeutic application of hematopoietic stem cells for lysosomal storage diseases.

 

 

The Paediatric Clinic-University of Milan-Bicocca, San Gerardo’s Hospital, Monza neonatal screening reference center.

 

 

 

 

The Stem Cells Lab and Research Center in Terni directed by Angelo Vescovi who collaborates with us on the research project for the therapeutic application of neural stem cells for lysosomal storage diseases.