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.

Our excellence in numbers

110

children treated with hematopoietic stem cell gene therapy

600

appointments per year

20

gene therapy/transplantations per year

We reach the highest goals for you

 Numerous local and international accreditations attest the extensive experience of the Department in the field.

JACIE Accreditation – Stem Cell Transplant Program

Ospedale San Raffaele

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JACIE Accreditation – Stem Cell Transplant Program

AIFA Phase I pediatric clinical trial center

Ospedale San Raffaele

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AIFA Phase I pediatric clinical trial center

European reference network for rare diseases (ERN) Rare Immunodeficiency, Autoinflammatory and Auto-Immune diseases (RITA)

Ospedale San Raffaele

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European reference network for rare diseases (ERN) Rare Immunodeficiency, Autoinflammatory and Auto-Immune diseases (RITA)

Certificate UNICEF-WHO BFH: Baby Friendly Hospital

Ospedale San Raffaele

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Certificate UNICEF-WHO BFH: Baby Friendly Hospital

World's Best Specialized Hospitals 2021 - 1st in Italy, 6th in the world

Ospedale San Raffaele

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World's Best Specialized Hospitals 2021 - 1st in Italy, 6th in the world

Research

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Where do we treat it?

At GSD Group, you can find this specialty at these departments: