15.11.24
Dawn's reflection: hope from Tigem research
The child, born with mucopolysaccharidosis type 6, was treated with gene therapy in 2020 and to date her liver is able to produce the missing enzyme that causes the disease.
11.10.24
A hope twelve thousand kilometres long
The story of Amari, who came from the small Caribbean Island of Trinidad to Milan to receive gene therapy for his rare genetic disorder of the immune system.
02.08.24
Violet's story: between research and outreach
The Canadian girl was born with mucopolysaccharidosis type 6. With help from the Isaac Foundation, she received important therapies in her country and participated in Italy in the trial developed by the Telethon Institute of Genetics and Medicine.
11.02.26
February 11: The Value of Women in Research
Bioinformatics and translational research at TIGEM, as told by two of its leading women researchers, on the International Day of Women and Girls in Science.
05.02.26
Gene therapy platform for lysosomal storage diseases
A new gene therapy platform for lysosomal storage diseases enables parallel development, reducing time, costs and improving sustainability for rare disease treatments.
16.01.26
Fondazione Telethon receives european marketing authorisation for Waskyra™ (etuvetidigene autotemcel), a gene therapy for the treatment of Wiskott-Aldrich syndrome
The European Commission’s decision follows the positive opinion issued by the EMA’s CHMP in November 2025. The therapy was also approved by the U.S. FDA for the U.S. market in December 2025.
