Patients
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.
17.12.22
Margaux discovering the world thanks to gene therapy
She was the first Belgian girl with the rare immunodeficiency ADA-SCID treated with the gene therapy approach developed by Telethon researchers. From the risk of a life of seclusion, she has conquered the possibility of doing whatever she likes.
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