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In a study presented at the annual ASH meeting, investigators link biomarkers of cardiopulmonary, renal, and liver dysfunction with specific outcomes for adult sickle cell disease patients.

An analysis of nearly 7000 health records led to insights into factors of fragmented care for patients with sickle cell disease.

The deoxygenated sickle hemoglobin polymerization inhibitor was granted Accelerated Approval designation for the burdensome rare disease.

Crizanlizumab is the first ever drug approved to reduce vaso-occlusive crises in patients with the rare blood disease.

Crizanlizumab is being developed to prevent painful and unpredictable vaso-occlusive crises in patients with sickle cell disease.

Models show that for every gram of hemoglobin increased per deciliter, the risks associated with hemolytic anemia decrease.

The therapy attempts to restore fetal hemoglobin production.

Familial Haploidentical Stem Cell Transplantation Improves Quality of Life & Neurocognition in Sickl
Long-term data shows familial haploidentical stem cell transplantation (HISCT) improves quality of life and neurocognition in sickle cell disease patients.

Treatment with crizanlizumab reduced pain in patients with sickle cell disease who were experiencing vaso-occlusive crisis.

The ID CORE XT is the second molecular assay approved for use in transfusion medicine and the first to report results as genotypes.

The National Institutes of Health has launched the Cure Sickle Cell Initiative to accelerate the pace at which genetic-based curative therapies are developed for sickle cell disease.

Investigators find boys treated with alkylating agents or with hydroxyurea for sickle cell disease resulted in a significant reduction in spermatogonial cell counts, suggesting that these agents can affect fertility.

The New England Journal of Medicine has published the 48-week phase 3 clinical trial results that supported the FDA approval of L-glutamine oral powder for the reduction of acute complications of sickle cell disease in adult and pediatric patients.

New findings suggest that a specific hemoglobin production protein could be a target for therapy in sickle cell disease or beta-thalassemia.

FDA Commissioner, Scott Gottlieb, MD, announced the FDA’s efforts to advance the development of gene therapies through the addition of 6 scientific guidance documents.

Stay up-to-date on the latest rare disease news by reading the top 5 articles of the week.

















































































