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.
03.03.25
Fondazione Telethon launches a platform dedicated to Duchenne Muscular Dystrophy
Created thanks to the contribution of some of the most important companies involved in the development of therapies for the disease, it will make available the data of hundreds of DMD patients and will lay the foundations for collecting new data in the future according to homogeneous standards.
03.02.25
Fondazione Telethon submits EU marketing authorization application for Etuvetidigene Autotemcel Gene Therapy for the treatment of Wiskott-Aldrich Syndrome
Fondazione Telethon announced that it has submitted the Marketing Authorization Application (MAA) for the gene therapy - etuvetidigene autotemcel - for the treatment of patients with Wiskott-Aldrich Syndrome (WAS), a rare genetic disease of the immune system to the European Medicines Agency (EMA).
03.12.24
ERC: two researchers from San Raffaele-Telethon institute for gene therapy in Milan each receive 2 million euros for their prestigious funding together with IRCCS Ospedale San Raffaele and Università Vita-Salute San Raffaele
Daniela Cesana and Alessio Cantore presented two projects, respectively to investigate the impact, safety and efficacy of gene therapy through the 'signatures' of circulating DNA and to investigate the biology of liver cells in order to exploit them in the field of gene therapy.
