News from Research

01 / 01

Gene therapy, especially if administered early, could be an effective treatment strategy for beta thalassemia, a genetic disease that is quite common in Mediterranean countries, affecting over 7000 patients in Italy only.

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.

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