Top-line data from the Phase 3 part of RISE UP, a clinical trial testing mitapivat in people with sickle cell disease (SCD), are expected by the end of the year, according to developer Agios Pharmaceuticals. If those results are positive and mitapivat wins regulatory approval for SCD, it…
The use of computed tomography pulmonary angiography (CTPA) to assess the presence of blood clots in lung blood vessels increased significantly in people with sickle cell disease (SCD) after 2020, a study in Bahrain shows. The study indicates that a third of patients underwent several scans, many of which…
More perceived social support from classmates is associated with less disease severity among children and adolescents with sickle cell anemia, the most common and often the most severe form of sickle cell disease (SCD), according to a new study from researchers in Nigeria. Social support from parents also was…
A toolkit may facilitate the active involvement of parents of young children with sickle cell disease (SCD) in shared decision-making regarding hydroxyurea treatment for their children, a study suggests. However, the odds of these young patients being offered and prescribed hydroxyurea were higher among those who received usual…
Hydroxyurea may effectively reduce the risk of stroke in children with sickle cell disease (SCD), a systematic review has found. “This review highlights the need for further research to understand the real-world feasibility and practical implications of hydroxyurea dosing strategies and to explore its long-term safety and efficacy…
A scientist at Florida Atlantic University, Sarah E. Du, has been named a senior member of the U.S. National Academy of Inventors in recognition of her significant contributions to innovation and invention, including her work on diagnostic and monitoring tools for sickle cell disease (SCD). The academy’s Senior Members…
Some children with sickle cell disease (SCD) may benefit from supplementing with folic acid, according to a clinical trial. Supplementation was found to significantly increase the levels of red blood cell folate, a molecule important for the production of red blood cells, compared with a placebo. However, no differences…
Vertex Pharmaceuticals has entered a reimbursement agreement with the National Health Service in England that will enable eligible sickle cell disease (SCD) patients in that country to get access to treatment with Casgevy (exagamglogene autotemcel) via the public health system. “[This] agreement means eligible sickle cell disease…
People with sickle cell disease (SCD) have brains that appear to be more than a decade older than their actual age. That’s according to a recent study that also found that patients with older-looking brains had poorer cognitive function. “Our study explains how a chronic illness … can cause…
Orna Therapeutics is partnering with Vertex Pharmaceuticals to develop gene-editing therapies for people with sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT). The three-year collaboration will leverage Orna’s proprietary lipid nanoparticle (LNP) delivery system. LNPs are tiny vesicles made up of fatty molecules that can be used…
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