Sickle Cell Disease

ORLANDO — Researchers identified the protein nuclear factor IX as a novel repressor of fetal hemoglobin, a discovery that could lead to the development of new treatments for sickle cell disease, according to data presented at ASH Annual Meeting and Exposition.

ORLANDO — Oral supplementation with arginine resulted in faster resolution of pain associated with vaso-occlusive episodes among younger patients with sickle cell disease, according to the results of a randomized phase 2 trial presented at ASH Annual Meeting and Exposition.

ORLANDO — Significant induction of fetal hemoglobin after the infusion of CD34-positive cells occurred among a small cohort of patients with sickle cell disease, according to data from a phase 1 gene therapy feasibility study presented at ASH Annual Meeting and Exposition.

ORLANDO — Young adults with sickle cell disease experienced fragmentation of health care during the transition period after childhood, according to the results of a retrospective study presented at ASH Annual Meeting and Exposition.

ORLANDO — Julie Kanter, MD, from the division of hematology-oncology at the University of Alabama-Birmingham, discussed the first-ever targeted FDA-approved drug for sickle cell disease, crizanlizumab, in a video at the ASH Annual Meeting and Exposition.

ORLANDO — Interim results of an international survey presented at the ASH Annual Meeting and Exposition indicate that complications of sickle cell disease, including vaso-occlusive crisis events as well as poor functioning and quality of life, may be underestimated.

The FDA granted accelerated approval to voxelotor for the treatment of sickle cell disease.

A moderate-intensity exercise regimen appeared to improve skeletal muscle microvasculature among patients with sickle cell disease, according to results of a randomized trial published in Blood.

The FDA approved crizanlizumab to reduce the frequency of pain crises among people with sickle cell disease.

September is National Sickle Cell Awareness Month.