Patricia Inácio, PhD, science writer —

The Institute for Clinical and Economic Review (ICER) says three recently approved therapies for managing sickle cell disease (SCD) are too costly based on early assessments of their effectiveness, and is recommending their prices be lowered. In a new evidence report, the independent watchdog agency on drug…

The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However, despite the increase, nearly 7,000 rare diseases are still lacking treatment. And federal incentives to boost treatment development for these rare diseases have primarily focused not on creating new…

White blood cells, especially monocytes, may underlie the breathing difficulties affecting children with sickle cell disease (SCD), a small pilot study suggests. Asthma is estimated to impact from 17% to 28% of all children with SCD, its researchers noted, and obstructive lung disease — which affects exhaling — is…

The National Institutes of Health (NIH) and the Bill & Melinda Gates Foundation will each invest $100 million over the next four years to speed the development of affordable gene therapies for sickle cell disease (SCD) and the human immunodeficiency virus (HIV) on a global scale.

Enhanced parenting skills, including attentive listening and engaging in conversations, may help improve cognitive functioning in children with sickle cell disease (SCD), a study suggests. Parent stress was related to lower parenting responsiveness, which also may lead to lower cognitive function in children, the researchers said. The study, “…

Caraphenol A — a small molecule closely related to a natural compound called resveratrol, which is produced by red grapes — increased the efficacy of hematopoietic stem cell-based gene therapy, a new study shows. The molecule holds potential to shorten the time and cost of using these gene therapies…

A newly designed viral vector — the vehicle that delivers a gene therapy to a patient’s cells — for use in sickle cell anemia is more efficient than earlier vectors at introducing healthy copies of genes into stem cells and can be produced in greater amounts, studies in animal models…

A community town hall is set for Detroit to raise awareness about sickle cell disease, with a discussion about current and future therapies, as part of the Sideline Sickle Cell campaign by Emmaus Life Sciences, together with the Sickle Cell Disease Association of America Michigan Chapter. The…

A medication used to treat high blood pressure, called valsartan, may help decrease the stickiness of red blood cells and reduce the risk of vaso-occlusive crisis in people with sickle cell disease (SCD), a research study suggests. The study, “Valsartan impedes epinephrine-induced ICAM-4 activation on normal, sickle cell…

An upcoming analysis by the Institute for Clinical and Economic Review (ICER) will compare the clinical efficacy and economic value of Novartis’ crizanlizumab and Global Blood Therapeutics‘ voxelotor, lead candidate therapies for sickle cell disease (SCD). ICER’s report will be discussed during its…