Blood levels of heme oxygenase-1, or HO-1, an enzyme that helps protect cells from certain types of damage, are elevated in people with sickle cell disease (SCD), especially children, a new study showed. HO-1 serves as a safeguard against the toxic effects of free heme, a molecule present in…
The U.S. Patent Office has given AB Science notice that it will allow a patent to be issued covering the use of the experimental therapy masitinib for sickle cell disease (SCD). This medical use patent will be valid until November 2040, giving AB Science exclusive rights to develop masitinib…
Most people with sickle cell disease (SCD) who received a new type of stem cell transplant in a clinical trial were free of disease-related clinical events after two years, according to a study. The scientists said the findings demonstrate that the approach, which uses a lower-intensity conditioning regimen…
A hematopoietic stem cell transplant (HCT) may be a feasible treatment option for adolescents and adults with severe sickle cell disease (SCD), according to a U.S. study. Two-year survival rates were similar among patients who had an available donor for HCT and among those who did not, although they were…
Fulcrum Therapeutics is planning to report, by midyear, new data from the ongoing PIONEER Phase 1b clinical trial that’s testing its oral therapy candidate pociredir (FTX-6058) in people with sickle cell disease (SCD), according to a new corporate presentation. That will include data from the study’s third-highest…
In a Phase 1/2 clinical trial, the gene-editing stem cell therapy BEAM-101 worked as intended, and showed early signs of efficacy in people with severe sickle cell disease (SCD), according to new early data. Developer Beam Therapeutics will present the findings at the upcoming American Society of Hematology…
Enlargement of the left atrium, one of the heart’s upper chambers, is associated with a greater sickle cell disease (SCD) burden for patients, with more emergency department visits and blood test abnormalities, according to a new study from the U.S. A larger left atrium is believed to be a…
People with sickle cell trait (SCT), those who carry a mutation in one copy of the HBB gene but do not have sickle cell disease (SCD), are at an increased risk of blood clots compared with people who don’t carry the trait regardless of racial or ethnic background, according…
A Massachusetts scientist has been awarded the seventh annual Trailblazer Prize for Clinician-Scientists by the Foundation for the National Institutes of Health (FNIH) for his research into understanding human blood cell development and how it’s altered in conditions such as sickle cell disease (SCD) — work that helped lead…
Seeking treatment for acute bouts of pain in hospitals and urgent care clinics is a significant source of distress for people with sickle cell disease (SCD), a report suggests. In interviews with SCD patients, scientists found patients’ distress went beyond the pain itself and included feelings of stigma and…
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