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.
23.06.26
ESMRank: using AI to predict the effects of protein mutations
At TIGEM, ESMRank uses artificial intelligence to rank protein mutations and help researchers interpret genetic variants linked to disease.
17.06.26
Doing a Postdoc in Advanced Therapies: why choose SR-Tiget?
At SR-Tiget, Postdocs are trained through excellence in research, a translational vision, mentorship, and international opportunities.
15.06.26
Fondazione Telethon appoints Graciana Diez-Roux as new Scientific Director
She will define the Foundation’s scientific strategy to further advance rare genetic disease research and support the next generation of scientists.
