Sickle Cell Disease news and research updates
Research readouts, treatment and access decisions, and community updates for people living with sickle cell disease and the people who care for them — each one dated, with a link to the original source where there is one.
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A Survey on Sickle Cell Disease Detection and Analysis
With increasing population numbers worldwide, sickle cell disease (SCD) continues to pose a serious global health problem, especially in sub-Saharan Africa, where close to 240,000…
Clonal hematopoiesis in the setting of sickle cell disease and its relevance to curative therapies
Abstract Sickle cell disease (SCD) is an inherited hemoglobinopathy characterized by chronic hemolytic anemia, painful vaso-occlusive episodes, and end organ damage. Population…
Orbital involvement in sickle cell disease: A systematic review
ABSTRACT Orbital involvement in sickle cell disease (SCD) is rare but potentially vision-threatening and often misdiagnosed due to overlap with infectious orbital disease. We…
Ten-Year Study Links Hydroxyurea Use to Reduced Mortality and Hospitalizations in Sickle Cell Patients
A ten-year study has identified significant clinical outcomes for patients with sickle cell anemia treated with hydroxyurea, including reduced mortality rates, fewer…
Life-changing benefits of hydroxyurea for sickle cell anemia affirmed by 10-year study
These are the benefits documented from 10 years of continuous hydroxyurea treatment provided in the NOHARM trial to a group of young children in Uganda, which has one of the…
The hidden struggle of Sickle Cell disease and the hope in new treatments
Sickle cell disease (SCD) is a group of inherited blood disorders that cause red blood cells to become hard, sticky, and crescent-shaped. Instead of flowing smoothly through blood…
Accurate identification of sickle cell disease cases in a large genotyped cohort using electronic health record data
Abstract The US Food and Drug Administration recently approved sickle cell disease (SCD) gene therapies that require a contemporaneous cohort to compare their outcomes with those…
Haploidentical transplant, gene therapy, and standard care in sickle cell disease: a cost-effectiveness analysis
Key Points Non-myeloablative haploidentical (NMAC-HID) transplant would be cost-effective for long-term SCD remission in adults Maximum gene therapy price versus standard care…
In Utero Gene Therapy for Sickle Cell Disease: Current Evidence, Ethical Considerations, and Future Directions—A Scoping Review
Abstract Purpose Sickle Cell Disease (SCD) is a monogenic, autosomal recessive disorder caused by mutations in the β-globin gene, resulting in the production of abnormal…
Elevated levels of hormone EPO tied to risk of blood clots in SCD: Large study
Higher blood levels of erythropoietin (EPO), a hormone that stimulates the production of red blood cells, increase the risk of blood clot-related, or thrombotic, events in people…
Fulcrum exploring a potential sale after FDA sidelined its sickle cell drug
Fulcrum Therapeutics has begun a strategic review that may result in it getting sold, a move made after the company’s main medicine hit a possibly insurmountable obstacle. On…
Fulcrum Halts Development of SCD Candidate Pociredir, Sets Strategic Review
Fulcrum Therapeutics said today it is scrapping its lead pipeline program to develop pociredir as a treatment for sickle cell disease (SCD) and launching a “comprehensive” review…