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Enzyvant gains double fast-track for rare disease drug
The investigational tissue-based therapy is for the treatment of complete DiGeorge Syndrome (cDGS), which represents a tiny subset of patients with the rare condition, DiGeorge Syndrome, caused by ...
Team links gene duplications, deletions within chromosome region to nonsyndromic bicuspid ...
DiGeorge syndrome, a 22q11.2 deletion syndrome, is caused when a small portion of chromosome 22 is missing. The condition can ...