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.
16.01.26
Fondazione Telethon receives european marketing authorisation for Waskyra™ (etuvetidigene autotemcel), a gene therapy for the treatment of Wiskott-Aldrich syndrome
The European Commission’s decision follows the positive opinion issued by the EMA’s CHMP in November 2025. The therapy was also approved by the U.S. FDA for the U.S. market in December 2025.
19.12.25
The sustainability of gene therapies for rare diseases: a model born from ADA-SCID and WAS
Fondazione Telethon have obtained marketing authorisation for a gene therapy thanks to a sustainability model that integrates research with industry, patients and supporters. Read the full interview with Celeste Scotti, R&D Director at Fondazione Telethon.
16.12.25
From Wiskott–Aldrich Syndrome to a new model for gene therapies
Rare and ultra-rare genetic diseases pose profound scientific, human and economic challenges. The development of gene therapy for Wiskott–Aldrich syndrome (WAS) is a clear example of this complexity.
