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With an estimated 1.37 billion inhabitants, India will likely surpass China in five years as the world’s most populous country. That also means it will have more rare-disease patients than any nation. It already has more than twice as many as the 28-member European Union. Harsha K. Rajasimha, a genomics…

In an effort to raise awareness about the latest in sickle cell disease therapy, Emmy award-winning National Football League (NFL) broadcaster Solomon Wilcots has partnered with biopharmaceutical company Emmaus Life Sciences to launch “Sideline Sickle Cell.” The educational campaign targets the sickle cell disease (SCD) community —…

Clinicians at the Loma Linda University Children’s Hospital successfully performed that institute’s first stem cell transplant, and report having “effectively cured” a girl with sickle cell disease (SCD). “She is now disease free and can…

Sickle cell disease patients experience silent strokes in the deep white matter of the brain that is prone to low oxygen supply, a brain-mapping study shows. The study, “Silent infarcts in sickle cell disease occur in the border zone region and are associated with low cerebral…

Abnormal sickle-shaped red blood cells characteristic of sickle cell disease (SCD) change the structure and dynamics of blood clots’ formations in veins, a study in mice and human blood shows. Such changes may explain why SCD patients are at higher risk for venous thromboembolisms. The study with those findings, “…

The Green Park Collaborative (GPC) is working with the Sickle Cell Disease Association of America (SCDAA) to develop a consensus of desired outcomes to be used in clinical studies evaluating new treatments for sickle cell disease (SCD). The project, named coreSCD, aims to deliver a list of recommendations on…

Consumers may know Pfizer as the company that manufactures Viagra, but the New York-based pharma giant is investing millions in developing potential treatments and gene therapies for rare disorders ranging from sickle cell disease and hemophilia to ALS and Duchenne muscular dystrophy. Michael Wajnrajch, MD, is senior medical director…

Children and adolescents with sickle cell anemia (SCA) showed low adherence to treatment with hydroxyurea, according to a multi-state study in the U.S. The research, “Hydroxyurea use among children with sickle cell anemia,” was published in the journal Pediatric Blood & Cancer. Hydroxyurea is…

Alterations in the lungs’ network of blood vessels, caused by sickle cell disease (SCD), reflect disease burden and should be carefully monitored over time, a recent study says. The study with that finding, “Clinical relevance of pulmonary vasculature involvement in sickle cell disease,” was published in the…

Development of problem-solving skills may help adolescents with sickle cell disease prepare for the transition to adulthood and also improve their ability to self-manage their health. Researchers at the Boston University Medical Center conducted a study to gain better insight on the perspectives of sickle cell disease (SCD)…