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.
02.03.26
Retinal dystrophies: gene-agnostic therapy based on miRNAs
New miR-based gene-agnostic strategies target shared mechanisms in retinal dystrophies, opening new therapeutic options beyond single-gene therapy.
27.02.26
Fondazione Telethon announces positive CHMP opinion recommending unlimited renewal of Strimvelis™ marketing authorisation, ten years after first approval
The ADA-SCID gene therapy, manufactured and distributed by Fondazione Telethon in the European Union since 2023, continues to demonstrate a favourable benefit-risk balance.
23.02.26
AAV gene therapy for large genes: two TIGEM platforms
How AAV gene therapy for large genes overcomes size limits through two TIGEM technology platforms for inherited retinal and rare diseases.
