Pediatric Immunohaematology
The Pediatric Immunohematology Department is a national and international reference center for the diagnosis of primary immunodeficiencies (PID) and other pediatric hematological diseases. Testifying the medical and scientific excellence of the San Raffaele’s Pediatric Immunohematology is its key role in the development and approval of the drug Strimvelis, the first lifesaving stem cell-based gene therapy drug in Europe, result of the strategic alliance between Ospedale San Raffaele, Telethon Foundation and GSK.
The Department is home to clinical trials of gene therapy, in collaboration with the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), for the treatment of beta-thalassemia, Wiskott-Aldrich syndrome, metachromatic leukodystrophy, and type I mucopolysaccharidosis. In collaboration with San Raffaele’s laboratories, there are also numerous researches related to genetic diagnosis for over 300 genes and to the study of the pathogenesis of congenital immunodeficiencies and genetic diseases with immunodysregulation.
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