New hope for ‘bubble boy’ disease

July 31, 2014 10:13 PM

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New hope for ‘bubble boy’ disease

Study in NEJM reviews 10 years of data, argues for expansion of newborn screening

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 the Harvard-affiliated Dana-Farber/Boston Children’s...

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