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…
A hospital with the University of Rochester’s Medical Center has received a multi-million-dollar grant to conduct a study comparing the efficacy of some available treatments for sickle cell disease (SCD), helping families to make better-informed treatment decisions. Particularly, the study by scientists with Golisano Children’s Hospital will compare…
AB Science said it received a notice of allowance from the European Patent Office for a patent covering the use of the experimental oral therapy masitinib to treat sickle cell disease (SCD). The decision was based on preclinical data demonstrating that masitinib was able to prevent vaso-occlusive crises…
A Nigerian hematologist will pursue a PhD in health policy at the University of Tennessee Health Science Center to help advance her work on programs for sickle cell disease (SCD) in her home country. Aisha Galadanci Akilu, MD, helped implement a newborn screening program in Kano City, Nigeria, which…
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