U.S. Department of Health & Human Services Divider Arrow National Institutes of Health Divider Arrow NCATS

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Status:
Designated

Class (Stereo):
CHEMICAL (ACHIRAL)


Isobutyramide (VX-366) is an orally available branched chain amide that may offer an alternative to current treatments for beta-hemoglobinopathy. Isobutyramide has been shown to increase fetal hemoglobin (HbF) in patients with beta-hemoglobinopathies...