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.
28.04.26
Fondazione Telethon launches Beyond, a new european postdoctoral program in advanced therapies
European Commission awards approximately €6 million under the Marie Skłodowska-Curie Actions, recognising Fondazione Telethon’s leadership in advanced cell and gene therapy research and training.
20.04.26
Fondazione Telethon’s genomic program ends the diagnostic odyssey for hundreds of children
Eight years of the Undiagnosed Disease Program by Fondazione Telethon: nearly 1 in 2 children receive a molecular diagnosis. A new study in Genetics in Medicine Open reports the main outcomes and describes a model that can be adopted by national health systems.
02.04.26
N-of-1 therapies: personalised treatments for single patients
How N-of-1 therapies and antisense oligonucleotides enable personalised treatments for ultra-rare genetic diseases, from mutation to clinical use.
