Expansion of Newborn Screening Could Improve Early Detection, Treatment of ‘Bubble Boy’ Disease

August 1, 2014

(Medical News Today) – Children born with so-called “bubble boy” disease have the best chance of survival if they undergo a hematopoietic stem cell transplant as soon after birth as possible, according to a detailed analysis of 10 years of outcome data by researchers at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center. These findings, the researchers say, argue for expanding newborn screening for severe combined immune deficiency (SCID), a disorder that leaves affected infants so vulnerable to infection that most die within the first year of life if untreated.