In children with sickle cell disease (SCD), ages 4–11, weight-based doses of Oxbryta (voxelotor) result in similar rises…
Articles by Marta Figueiredo, PhD
The Saudi Food and Drug Authority (SFDA), in Saudi Arabia, will review Emmaus Life Sciences’ application for the approval…
Six months of treatment with Oxbryta (voxelotor) safely and effectively increased hemoglobin levels and reduced red blood cell destruction,…
The U.S. Food and Drug Administration (FDA) has expanded the label of Chiesi Global Rare Diseases’ iron-binding oral treatment…
People with sickle cell anemia have higher levels of mitochondrial DNA (mtDNA) — DNA specific to mitochondria, the cell’s powerhouses…
Treatment with oral Oxbryta (voxelotor) leads to rapid and sustained rises in hemoglobin levels, reduces red blood cell destruction (hemolysis),…
The U.S. Food and Drug Administration approved the start of a first clinical trial of CRISPR_SCD001, the first non-viral and…
People with rare disorders have a worse healthcare experience than those affected by chronic diseases, according to the results of…
A good diet and companionship, or treatment with an antidepressant called duloxetine was seen to significantly lessen pain in a…
A new National Heart, Lung, and Blood Institute (NHLBI) challenge will award a first prize of $25,000 to a team…