At the San Raffaele-Telethon Institute in Milan researchers are working to apply gene therapy, an approach that has already given important results, to even more diseases optimizing development times and costs.
Tag Archives: Istituto San Raffaele Telethon per la terapia genica
Margaux discovering the world thanks to gene therapy
She was the first Belgian girl with the rare immunodeficiency ADA-SCID treated with the gene therapy approach developed by Telethon researchers. From the risk of a life of seclusion, she has conquered the possibility of doing whatever she likes.
Orchard Therapeutics Receives EC Approval for Libmeldy™ for the Treatment of Early-Onset Metachromatic Leukodystrophy (MLD)
First gene therapy to receive full EU marketing authorization for eligible MLD patients. One-time treatment with Libmeldy has been shown to preserve motor and cognitive function. Achievement shared with research alliance partners Fondazione Telethon and Ospedale San Raffaele.