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AbstractThe US Food and Drug Administration recently approved sickle cell disease (SCD) gene therapies that require a contemporaneous cohort to compare their…

Key Points Non-myeloablative haploidentical (NMAC-HID) transplant would be cost-effective for long-term SCD remission in adultsMaximum gene therapy price…

AbstractPurposeSickle Cell Disease (SCD) is a monogenic, autosomal recessive disorder caused by mutations in the β-globin gene, resulting in the production of…

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,…

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…

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 “…

SummaryQuestionThis study analyzed the cost-effectiveness of three treatment strategies for sickle cell disease (SCD): standard care (SOC), gene therapy (GT), and…

June 2 (Reuters) - Shares of Fulcrum Therapeutics (FULC.O), opens new tab plunged 52% on Tuesday after the company said it would abandon development ​of its…

CorrectSequence Therapeutics Reports Positive 15-Month Follow-Up Data for CS-206SHANGHAI, June 2, 2026 /PRNewswire/ -- CorrectSequence Therapeutics Co., Ltd. (…

The drug, pociredir, was being developed as an ​oral treatment for sickle-cell disease, an inherited blood disorder that can cause ​pain, anemia, organ damage and…