From Telethon Foundation
14.11.25
CHMP positive opinion for Waskyra™, a gene therapy for the treatment of Wiskott-Aldrich syndrome (WAS)
Announced the positive opinion issued by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA), recommending marketing authorisation in the European Union for Waskyra™, an ex vivo gene therapy for Wiskott-Aldrich Syndrome.
16.06.25
Andrea Ballabio awarded Beth Levine Prize in Autophagy Research from UT Southwestern
Andrea Ballabio has been named the recipient of the 2025 Beth Levine, M.D. Prize in Autophagy Research from UT Southwestern Medical Center.
11.03.25
BLA for the gene therapy for the treatment of Wiskott-Aldrich syndrome submitted to FDA
The Biologic License Application for the gene therapy for the treatment of the Wiskott-Aldrich Syndrome (WAS) has been submitted to the Food and Drug Administration
03.03.25
A platform dedicated to Duchenne Muscular Dystrophy
Created thanks to the contribution of some companies, 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
MAA for the Gene Therapy for the treatment of Wiskott-Aldrich Syndrome submitted to EMA
The Marketing Authorization Application for the gene therapy for the treatment of Wiskott-Aldrich Syndrome (WAS) has been submitted to the European Medicines Agency
05.09.24
ERC Funding to three young researchers from Fondazione Telethon Institutes
Fondazione Telethon has announced that the European Research Council, has awarded three researchers from Fondazione Telethon Institutes: Samuele Ferrari and Attya Omer from SR-TIGET and Ivana Trapani from TIGEM
10.07.24
Ilaria Villa new Managing Director of Fondazione Telethon
Fondazione Telethon Board of Directors chaired by Luca di Montezemolo appoints Ilaria Villa as new Managing Director.
15.04.24
Alberto Auricchio appointed as director at TIGEM
Alberto Auricchio, full professor of Medical Genetics at the Federico II University of Naples, is the new director of the Telethon Institute of genetics and Medicine
28.02.24
Fondazione Telethon announces commitment to make gene therapy for Wikott-Aldrich Syndrome available to patients
The announcement arrives on the eve of the 26th World Rare Disease Day
