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.
16.06.25
Andrea Ballabio awarded Beth Levine Prize in Autophagy Research from UT Southwestern
Andrea Ballabio, founder and former director of the Telethon Institute of Genetics and Medicine (TIGEM) and Professor of Medical Genetics at the University of Naples “Federico II” has been named the recipient of the 2025 Beth Levine, M.D. Prize in Autophagy Research from UT Southwestern Medical Center.
24.04.25
MLD and gene therapy: a game-changing treatment backed by NEJM—Timing Is Everything
The study was conducted at Ospedale San Raffaele in Milan and is the result of more than 20 years of research by the San Raffaele-Telethon Institute for Gene Therapy and the commitment of Fondazione Telethon to transforming research into therapies available for patients.
31.03.25
From psoriasis to Lowe syndrome: clinical trial kicks off thanks to collaboration between Fondazione Telethon and Can-fite
Research conducted by Antonella De Matteis at TIGEM has identified a molecule able to counteract renal dysfunction in this rare syndrome of genetic origin.
