A patient with severe sickle cell disease (SCD) remained free of two key SCD symptoms — including painful vaso-occlusive crises — for more than a year after receiving a single dose of CS-206, Correctsequence Therapeutics‘ investigational gene-editing therapy, new data show. The woman represents the first patient…
News
Stem cell transplant from a partially matched donor is more cost-effective than gene therapy to treat sickle cell disease (SCD), according to a study. In order for currently available gene therapies to deliver cost effectiveness on par with stem cell transplant, their cost would need to come down…
Fulcrum Therapeutics said it is discontinuing development of pociredir, its experimental oral treatment for sickle cell disease (SCD), after the U.S. Food and Drug Administration (FDA) raised concerns that the therapy may increase the risk of certain blood cancers. The concerns stem from the unexpectedly high rate 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, events in people with sickle cell disease (SCD), according to a study involving more than 500 patients. Researchers also discovered a relationship between the production…
The use of pharmaceutical disease-modifying therapies (pDMTs) has increased in recent years among both children and adults with sickle cell disease (SCD), according to a study analyzing U.S. claims data. Over the same eight-year period, the use of chronic blood transfusion therapy (CBT) has remained low and has…
Higher vitamin A levels in the blood were significantly associated with better lung function in children and adolescents with sickle cell anemia (SCA), the most common and severe form of sickle cell disease (SCD), a small study shows. Given that vitamin A levels tend to be low in children with…
An experimental, single-dose gene-editing therapy from Tessera Therapeutics successfully corrected the genetic mutation that causes sickle cell disease (SCD) in the majority of blood-forming stem cells in nonhuman primates, reaching levels that surpass what is expected to cure the disease. The new preclinical data were presented by Tessera…
Nearly every person with sickle cell disease (SCD) who underwent a new regimen for stem cell transplant, developed by doctors at Johns Hopkins in the U.S., is alive and has been free of disease activity in the years following the procedure, a study reports. “The survival rate…
Agios Pharmaceuticals has filed an application with the U.S. Food and Drug Administration (FDA) seeking accelerated approval of mitapivat, its oral therapy for sickle cell disease (SCD). The developer said it expects to receive notice of acceptance of the application and the anticipated review timeline following the FDA’s…
Newer oral anticoagulants, or blood thinners, were linked to a lower risk of clinically relevant nonmajor bleeding than warfarin, an older blood thinner, in adults with sickle cell disease (SCD) treated for dangerous blood clots, according to a study led by researchers in Saudi Arabia. However, no significant differences…
Recent Posts
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- Elevated levels of hormone EPO tied to risk of blood clots in SCD: Large study
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