A phase 2 trial of HQK-1001 in HbE-β thalassemia demonstrates HbF induction and reduced anemia

Blood. 2014 Mar 20;123(12):1956-7. doi: 10.1182/blood-2013-11-538470.
No abstract available

Publication types

  • Clinical Trial, Phase II
  • Letter
  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Butyrates / administration & dosage
  • Butyrates / adverse effects
  • Butyrates / therapeutic use*
  • Female
  • Fetal Hemoglobin / biosynthesis*
  • Hemoglobin E / genetics*
  • Hemoglobins / metabolism
  • Humans
  • Male
  • Young Adult
  • beta-Thalassemia / blood*
  • beta-Thalassemia / drug therapy*
  • beta-Thalassemia / genetics

Substances

  • Butyrates
  • Hemoglobins
  • Hemoglobin E
  • Fetal Hemoglobin
  • 2,2-dimethylbutyric acid