Patients' stories

01 / 02

Tommaso was treated for ADA-SCID in February 2016 at the San Raffaele Telethon Institute in Milan (SR – Tiget).

News from Research

02 / 02

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 therapy recommended for EU approval for eligible patients with confirmed diagnosis of late infantile or early juvenile MLD variants.

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