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Landmark study shows gene therapy a successful treatment for some with bubble boy syndrome

  TORONTO A new landmark study examining treatment for a debilitating genetic disease known colloquially as bubble boy syndrome has found that gene therapy may provide a solution. There are several types of severe combined immunodeficiency due to adenosine deaminase (SCID or ADA-SCID), but “bubble boy syndrome” refers to when affected children must be extra cautious and protected from germs and infections as their immune systems do not function properly. The study followed 50 patients, 20 from the U.K. and 30 in the U.S. – including eight patients from Canada who had been referred by specialists to take part of the study south of the border.

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