First U.S.-treated patient doing well!

Agustín has a form of X-linked Severe Combined Immunodeficiency (SCID-X1), better known as “bubble boy disease.” It affects only boys, leaving their bone marrow unable to make T-lymphocytes—white blood cells that fight infection. But now, after participating in a gene therapy trial at Children’s Hospital Boston, Agustín’s T cell function has tested normal.

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