Rare Disease Report - Researchers at St. Jude Children’s Research Hospital have developed a new gene therapy that creates fully-functioning immune systems in babies diagnosed with severe combined immunodeficiency (SCID), commonly referred to as the “Bubble Boy” disease.
This extremely rare condition is the effect of a genetic mutation and impacts 1 in every 50,000 to 100,000 live births, with patients almost always being male. Patients with SCID are unable to produce T-cells, B-cells and natural killer cells that protect the body from viruses and bacteria.
Per St. Jude, symptoms commonly include recurrent, persistent infections, rashes, diarrhea, fever, ear infections, and pneumonia. Children are also unable to gain weight or grow at a normal pace...>>>