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Gene Therapy for X-linked Chronic Granulomatous Disease (CGD) (NET4CGD)

Start date: Dec 1, 2012, End date: May 31, 2018,

This project is focused on the clinical development of a new orphan drug that can rapidly become a new treatment option for patients with the X-linked form of chronic granulomatous disease (X-CGD). This rare primary immune deficiency of phagocytes is caused by mutations in the gp91phox gene. Affected patients are highly-susceptible to infections and develop inflammatory granulomas. Several member ...
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