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.
29.05.26
Fondazione Telethon advances national Duchenne registry for research and regulation
A nationwide network of 16 Italian neuromuscular centers and over 1,200 patients to support research and new therapies.
19.05.26
SR-Tiget’s Development Facilities: where gene therapy takes shape
How SR-Tiget’s development facilities are key to turn research into advanced therapies, and why they represent a new professional frontier.
08.05.26
Lysosome Biology: From “Suicide Bag” to Metabolic Hub
Tigem research reveals the lysosome as a metabolic hub via TFEB and mTOR, enabling new treatments for lysosomal diseases, neurodegeneration and cancer.
