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.
15.06.26
Fondazione Telethon appoints Graciana Diez-Roux as new Scientific Director
She will define the Foundation’s scientific strategy to further advance rare genetic disease research and support the next generation of scientists.
10.06.26
TUDP: genomics, bioinformatics and knowledge for undiagnosed diseases
Fondazione Telethon’s TUDP combines genomics, bioinformatics and research to identify the genetic causes of rare paediatric diseases without a diagnosis.
03.06.26
New “SMArT” platform makes gene editing in hematopoietic stem cells more efficient and safer
Nature Biotechnology study shows near-pure selection of correctly edited blood stem cells while reducing dangerous genomic alterations.
