News

Ahead of this year’s Rare Disease Week on Capitol Hill, held virtually July 14–22, the EveryLife Foundation will award grants to top advocates of rare disease organizations who participate in the week’s pre-events. The top 50 point-earners will be eligible to win $1,000 to $5,000in  grants, totaling up…

Machine learning tools can identify sickle cell disease (SCD) patients at high risk of progressive kidney disease as early as six months in advance, a study shows. The study, “Using machine learning to predict rapid decline of kidney function in sickle cell anemia,” was published in the journal…

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…

Rapid and repeated imaging of blood flow through the eyes can inform physicians of a patient’s risk of sustaining eye damage and developing vision problems related to sickle cell disease (SCD), as well as of how effective a given therapy is, a recent study shows. According to researchers, this…

ARU-1801, an experimental gene therapy for sickle cell disease (SCD), lowered the number of vaso-occlusive crises and led to  blood cells producing fetal hemoglobin in a first group of three treated patients, according to data from an ongoing Phase 1/2 trial. Favorable safety is…

Two U.S. nonprofits are partnering to tackle legislative issues that benefit sickle cell disease (SCD) patients and their families. The partnership between the Sickle Cell Disease Association of America (SCDAA) and the Sickle Cell Community Consortium (SCCC) will include collaborating on the association’s annual Advocacy Day…

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…

A non-viral, CRISPR-based gene-editing tool showed a potential to reverse mutations that cause sickle cell disease (SCD) by successfully editing genes in stem cells within the bone marrow of mice. Data from this proof-of-concept study…

A spoken word poetry contest is welcoming submissions from people with sickle cell disease (SCD) and their caregivers. The contest, “Lift Every Voice to Shine the Light on Sickle Cell,” is being jointly launched by Global Blood Therapeutics (GBT) and the Sickle Cell Disease Association of America…

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…