Stories
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.
31.05.22
In Italy for love and by choice
The story of Attya and her personal and professional journey that took her to the San Raffaele Telethon Institute for Gene Therapy in Milan.
10.10.19
Lorenzo wants to go far
“I can’t walk”. When you hear those words on the lips of a child below the age of three, it’s hard to turn your back and walk away. Imagine you’re the child’s parents. The words came out with the composed calmness of a fact of nature.
10.10.19
Against mitochondrial diseases together with Andrea
Knowing that the machine you need to breathe with has just one hour’s run time doesn’t make life very easy!
09.10.19
Gabriele's world
Gabriele is fast! Faster than the speed of light! On his “spiderina” – his little ‘sports car’ – he runs circles around his mum and dad, Daniela and Francesco!
27.11.18
Tommaso and his return to Normality
Tommaso was treated for ADA-SCID in February 2016 at the San Raffaele Telethon Institute in Milan (SR – Tiget).
