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.
29.09.26
New gut-liver link discovered in alpha-1 antitrypsin deficiency
A study led by Nunzia Pastore at TIGEM shows that the mutated protein responsible for the disease also alters intestinal cells, opening new avenues for identifying at-risk patients and understanding the variability of the disease.
28.09.26
UCSF Benioff Children’s Hospitals Become the First US Qualified Treatment Center Contracted to Administer Individualized Gene Therapy WASKYRA®
WASKYRA®, the first gene therapy approved in the US to treat Wiskott-Aldrich Syndrome, is now commercially available, with more centers expected to be added in the coming months.
24.09.26
Wiskott-Aldrich Syndrome: Long-Term Results of Gene Therapy Developed at the San Raffaele-Telethon Institute Published in the “New England Journal of Medicine“
The publication consolidates more than two decades of research and clinical development and confirms the long-term clinical benefit of an autologous gene therapy for Wiskott-Aldrich syndrome, a rare genetic disorder affecting the immune system and platelets.
