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Myelodysplastic syndrome (MDS) is a group of disorders characterized by dysfunctional blood cells that can progress to acute myeloid leukemia (AML). At this time the etiology of MDS development and progression to AML is not understood and there is no cure (median survival at time of diagnosis is less than 5 years). Yet, DNA damage and mutations appear to play a key role with the initiating defect in a hematopoietic stem cell (HSC). In support, people with certain DNA repair defective syndromes including Fanconi anemia (FA) and Bloom syndrome (BS) exhibit MDS.

UT Health San Antonio
Hasty, Edward Paul

This is a Phase 3, randomized, double-blind, placebo-controlled, multicenter study to evaluate the efficacy and safety of 3 treatment regimens of oral GED-0301 versus placebo in subjects with active CD (defined by a CDAI score ? 220 and ? 450 and a total SES-CD ? 6 at screening, or the ileum segmental SES-CD ? 4 at screening).

Brooke Army Medical Center
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