News

The use of eculizumab, an immunosuppressive medication approved for several autoimmune diseases, was found to be safe and effective for treating hyperhemolysis syndrome or HHS — a serious complication of blood transfusions — in two pediatric patients with sickle cell disease (SCD), a study showed. HHS, which can be…

Women with sickle cell disease who undergo a stem cell transplant can become pregnant and give birth to healthy children, a new study shows. “The results of this study show that female [stem cell transplant] recipients can achieve successful and safe pregnancies,” Katja Sockel, MD, lead study author at University…

Emmaus Life Sciences, which markets Endari (L-glutamine) for sickle cell disease (SCD), has announced that it is complying with postmarketing safety regulations of the U.S. Food and Drug Administration (FDA). The announcement followed a 2.5-day routine inspection of Emmaus under the FDA’s Postmarketing Adverse Drug Experience…

Seeking treatment for acute bouts of pain in hospitals and urgent care clinics is a significant source of distress for people with sickle cell disease (SCD), a report suggests. In interviews with SCD patients, scientists found patients’ distress went beyond the pain itself and included feelings of stigma and…

Supplementing with vitamin D for six months safely improved bone health and functional capacity in children and adolescents with sickle cell disease (SCD), a study finds. More studies are required to assess the “optimal dose, duration of supplementation, long-term [adverse events], and efficacy in different types of SCD, wrote…

The probability of developing organ and tissue damage — including in the lungs, kidneys, or eyes — increases as teenagers with sickle cell disease (SCD) transition from adolescence into adulthood, according to a study by researchers in France. Particularly, the study demonstrated that disease morbidity associated with chronic organ…

TALEN, a gene-editing technology being used by the clinical-stage biopharmaceutical company Cellectis to develop potentially life-changing therapies, was able to correct the mutation that causes sickle cell disease (SCD) in patient cells. That’s according to a new study published by researchers from the company, along with colleagues from…

All 18 adults with severe sickle cell disease (SCD) treated with the gene-editing therapy renizgamglogene autogedtemcel — known as reni-cel — remained free of vaso-occlusive events, or VOEs, for up to nearly two years, according to new interim data from the Phase 1/2/3 RUBY clinical trial. The therapy, being…

Nearly all people with sickle cell disease (SCD) treated with the U.S.-approved gene-editing therapy Casgevy (exagamglogene autotemcel) were free of painful vaso-occlusive crises (VOCs) for at least one year, according to new data from a Phase 2/3 clinical trial. These updated results were presented at the annual…

The Sickle Cell Disease Foundation (SCDF), in partnership with the Center for Inherited Blood Disorders (CIBD) and The Hills Tandem, is leading a real-world health data initiative that is progressing toward its goal of improving sickle cell disease (SCD) patients’ life quality and transforming their care. The…