M. Louise Markert – Complete DiGeorge Anomaly

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Complete DiGeorge anomaly (cDGA) is a disorder in which there is no thymus function. With no
thymus function, bone marrow stem cells do not develop into T cells, which fight infection.
Complete DiGeorge anomaly patients cannot fight infection and are immunodeficient. Without
successful treatment, cDGA patients usually die by age 2 years.

Thymus transplantation with and without immunosuppression (drugs given before and after
transplantation) has resulted in the development good T cell function in complete DiGeorge
anomaly subjects.

This Phase I/II study continues thymus transplantation safety and efficacy research for the
treatment of complete DiGeorge anomaly. Eligible participants undergo thymus transplantation
and biopsy. Immune function testing is continued for one year post-transplantation.

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