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5-Amino-1MQ is designed precisely for this phenotype

The primary endpoint was progression-free survival (PFS)

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By contrast, in studies including patients with SCA, BCL11A levels and SNPs were not found to be correlated with HbF induction after HU treatment [58], [59], [60], suggesting an important role for BCL11A in controlling baseline HbF levels, but with other factors required for HU to increase HbF levels