Tommaso was born in January 2015 in Rome to his mother Marta and father Andrea. One night, when he was two months old, he had such a bad case of flu that he was hospitalised for tests.

Tommaso and his father, Andrea

The diagnosis came 3 months later: ADA – SCID. Doctor Caterina Cancrini of the Bambino Gesù Children’s Hospital sent the family to the San Raffaele Telethon Institute in Milan (SR – Tiget), where Tommaso was treated in February 2016. Marta and Andrea have great faith in the work carried out by Professor Aiuti and his staff, and in view of the results obtained on other children treated, they are very optimistic. Their hope is that gene therapy will allow Tommaso to lead a normal life, spend time with other children and play with his cousins, whom he has hardly ever seen.

Tommaso appears just like any other little boy: cheerful, lively, always smiling, always on the move. Before she found out about Tommaso’s condition, Marta worked with children, and was always at risk of picking up flu bugs and other illnesses common among nursery school children, so, in order to protect Tommaso’s health, she has had to give up her job. Their life has to be strictly organised at all times: even just for a trip to the supermarket, Marta has always had to take Tommaso to his grandparents first, because he has never been able to have contact with other children, or spend any time indoors in busy places.

Until recently, the only curative treatment for ADA-SCID was a bone marrow transplant, which could be carried out only if a compatible donor was available. In 2002, researchers from the Telethon Institute in Milan demonstrated, for the first time, the efficacy of gene therapy, which in the years since then has proved to be safe, with lasting efficacy.

Thanks to an agreement signed in 2010 with the multinational pharmaceutical company GlaxoSmithKline, gene therapy is available on the market today and accessible to all patients in Europe.

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