FOR THESE PATIENTS, EVERY DAY IS A VICTORY IN ITSELF

Some patients travel miles to reach us. Giovanni and his family arrived from the United States. He suffers from metachromatic leukodystrophy (MLD). His sister, Liviana had the same disease. She is now among the angels. Thanks to her, however, it immediately became clear that her little brother had the same terrible disease. Giovanni is well, as are his two younger siblings, Ella and Eliah. Jacob also suffers from the Wiskott-Aldrich syndrome. Widad has ADA-SCID.

Gene therapy is vital for kids presenting such genetic diseases as these, for whom there was no hope prior to this (100% Italian) breakthrough. For two decades now, Telethon has provided funding for top class research in this field, fully aware of the potentials of such activities. The results tell us we were justified in making this decision! The list of diseases potentially curable with gene therapy is constantly being updated. The clinical study for thalassaemia has just started up. The type I and VI mucopolysaccharidosis study is ready to start up during this coming year. Other studies involving haemophilia, the Stargardt syndrome or the Usher syndrome may perhaps be started up over the next few years. We won’t give up the fight! Thanks to our volunteers and researchers − and thanks also to the generosity of the Italians − we’ll be able to write that word ‘curable’ next to the names of many more diseases of this kind! Tiget, in Milan, a world leader in the field of gene therapy The therapy developed at Tiget against ADA-SCID (a rare primitive immunodeficiency condition) is one of the world’s first instances of efficacious gene therapy. This result was made possible thanks to the far-sightedness of our organisation, which immediately expressed its belief in the benefits of gene therapy.

SR-Tiget, in Milan, is a world leader for gene therapy

The therapy developed at SR-Tiget against ADA-SCID (a rare primitive immunodeficiency condition) is one of the world’s first instances of efficacious gene therapy. This result was made possible thanks to the far-sightedness of our organisation, which immediately expressed its belief in the benefits of gene therapy.