The sickle disease of 83 percent of adolescents improved after 16 weeks of taking Global Blood Therapeutics‘ voxelotor, preliminary results of a Phase 2a clinical trial indicate. Another important finding was that voxelotor prevented irregularly shaped oxygen-carrying hemoglobin cells from clumping in more than half of those treated. The clumping prevents…
Researchers at Mississippi State University (MSU) are using tiny polymer chips to build models to help them better understand sickle cell disease. Research on the models, which can also be used to study heart disease, might lead  identification of molecules or factors that new treatments can target to lessen organ damage…
A 13-year-old boy with sickle cell disease has become the world’s first patient to be successfully treated with gene therapy. The unidentified teenager remains free from sickle crises and other signs of the disease 15 months after his treatment, which was conducted as part of a Phase 1/2 clinical trial…
Intensifying treatment with hydroxyurea in young children with sickle cell anemia is safe and improves several disease parameters in the long run, researchers demonstrated in a clinical trial (NCT00305175) comparing toddlers with older children. Based on the positive findings, researchers argue that the practice of giving small children higher…
Removing the spleen by keyhole surgery is a feasible option for children with sickle cell disease, according to a study that explored the procedure as the sole therapeutic intervention. But because the study described only short-term outcomes, researchers underscored that more research is needed to investigate how a splenectomy affects…
Researchers were able to mend the mutated gene that causes sickle cell disease, and successfully transfer the corrected stem cells into mice. Their work could have enormous implications for millions worldwide, as it is proof-of-concept that such a treatment might also work in humans. The study, “CRISPR/Cas9 β-globin gene targeting in human haematopoietic…
In a discovery that appears to turn textbook knowledge on its head, researchers at Massachusetts General Hospital and Harvard Medical School found that damaged or old red blood cells — and the iron they carry — are in fact mainly taken care of by the liver and not, as previously…
Women with sickle cell disease have a high risk of complications during pregnancy, according to new research from French Guiana. The findings also show that women with the more severe SS-type sickle cell disease are more likely to have premature babies, and to suffer health problems themselves, than women with…
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