Marta Figueiredo, PhD, science writer —

In children with sickle cell disease (SCD), ages 4–11, weight-based doses of Oxbryta (voxelotor) result in similar rises in hemoglobin levels and reductions in red blood cell destruction (hemolysis) to those observed in older patients at the approved dose, six-month data from the ongoing Phase 2 HOPE-KIDS 1…

The Saudi Food and Drug Authority (SFDA), in Saudi Arabia, will review Emmaus Life Sciences’ application for the approval of its oral medication Endari (L-glutamine) for treating sickle cell disease (SCD). Given that the SFDA’s review process typically takes 12 to 18 months, a decision is not expected before…

Six months of treatment with Oxbryta (voxelotor) safely and effectively increased hemoglobin levels and reduced red blood cell destruction, or hemolysis, in children ages 4–11 with sickle cell disease (SCD), according to data from the ongoing Phase 2 HOPE-KIDS 1 trial. These findings are consistent with those…

The U.S. Food and Drug Administration (FDA) has expanded the label of Chiesi Global Rare Diseases’ iron-binding oral treatment Ferriprox (deferiprone) to include patients ages 3 and older who have blood transfusion-induced iron overload due to sickle cell disease (SCD) or other anemias. The therapy was first approved…

People with sickle cell anemia have higher levels of mitochondrial DNA (mtDNA) — DNA specific to mitochondria, the cell’s powerhouses — circulating in the blood than healthy individuals, likely due to the abnormal retention of mitochondria in red blood cells, a study has found. Notably, patients’ mtDNA was found to…

Treatment with oral Oxbryta (voxelotor) leads to rapid and sustained rises in hemoglobin levels, reduces red blood cell destruction (hemolysis), and improves overall health in adolescents and adults with sickle cell disease (SCD), according to full, nearly 1.5-year data from the Phase 3 HOPE clinical trial. The findings support the long-term…

The U.S. Food and Drug Administration approved the start of a first clinical trial of CRISPR_SCD001, the first non-viral and CRISPR/Cas9-based gene editing therapy for sickle cell disease (SCD). Both the therapy and the upcoming Phase 1/2 trial — planned to start this summer — are the result of a…

People with rare disorders have a worse healthcare experience than those affected by chronic diseases, according to the results of an international survey conducted by Eurordis-Rare Diseases Europe. Indeed, rare disease patients overall give their healthcare experience a medium-low rating, of 2.5 on a scale of 1 to 5,…

A good diet and companionship, or treatment with an antidepressant called duloxetine was seen to significantly lessen pain in a mouse model of sickle cell disease (SCD), a study reports. Notably, each intervention was associated with higher levels in the animals of serotonin, a brain chemical messenger involved in stabilizing…

A new National Heart, Lung, and Blood Institute (NHLBI) challenge will award a first prize of $25,000 to a team of college or graduate students who develop an innovative tool to help spread evidence-based information about sickle cell disease (SCD). A total of $50,000 will be granted to the three…