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…
The European Commission has granted orphan medicinal product status to mitapivat, Agios Pharmaceuticals’ oral investigational therapy for sickle cell disease (SCD). This designation is granted to treatments for rare diseases, or those affecting fewer than five in 10,000 people in the European Union, that have the potential…
Nearly all the patients with severe sickle cell disease (SCD) who were treated with renizgamglogene autogedtemcel (reni-cel) remained free of vaso-occlusive events (VOEs) for up to two years, new data from the Phase 1/2/3 RUBYÂ clinical trial shows. The treatment also increased total hemoglobin levels and was well tolerated,…
Etavopivat, Novo Nordisk’s investigational oral therapy for sickle cell disease (SCD), may reduce the incidence of vaso-occlusive crises (VOCs) and increase hemoglobin levels. That’s according to data from the phase 2 part of the Phase 2/3 HIBISCUS trial (NCT04624659), which is assessing the treatment’s safety and…
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