Memantine, an oral medication that’s been used for decades to help treat Alzheimer’s disease, is generally tolerated well by people with sickle cell disease (SCD) and may help prevent painful disease crises, especially in children. Those are the findings of a small, one-year study in Israel that tested…
News
The breakdown of red blood cells — a defining feature of sickle cell disease (SCD) — may contribute to the cognitive problems many people with the condition experience, a study suggested. Researchers found that the breakdown of red blood cells, known as hemolysis, releases heme into the bloodstream, which…
Higher levels of fetal hemoglobin (HbF) seem to protect people with sickle cell disease (SCD) by reducing the risk of lung problems, such as pulmonary hypertension, and delaying bone damage, at least in men, according to a study from a single center in Georgia. The study, “The Role…
In a mouse study, blood stem cells from sickle cell disease (SCD) patients modified with CRISPR/Cas9 gene editing showed reduced long-term contribution compared with cells treated using other gene-based approaches. “While all methods showed therapeutic potential, base editing and [gene therapy] provided superior outcomes over CRISPR-Cas9-mediated editing in a…
Emmaus Life Sciences has entered into an agreement to sell the North American rights to Endari (oral L-glutamine), its oral treatment to prevent acute complications related to sickle cell disease (SCD), to Neoimmunetech. Under the agreement, Neoimmunetech will receive an exclusive license to all rights for marketing,…
Hydroxyurea, blood transfusions, and donor blood stem cell transplants can significantly lower the risk of stroke in children and adolescents with sickle cell anemia (SCA), the most common and severe form of sickle cell disease. That’s according to a systematic review and meta-analysis of 40 studies that also highlighted…
Big Nova has made a $3 million donation to support NMDP’s efforts to help more patients, including those with sickle cell disease (SCD), access blood stem cell transplants. The philanthropic gift helped NMDP exceed its $100 million fundraising goal, a milestone the organization said will support its…
A high dose of pociredir, Fulcrum Therapeutics’ investigational oral therapy for sickle cell disease (SCD), was associated with increased fetal hemoglobin (HbF) levels and reduced markers of red blood cell destruction in early trial data. Those preliminary findings come from a Phase 1b clinical trial called PIONEER…
Risto-cel, Beam Therapeutics‘ single-dose, gene-edited cell therapy candidate for people with severe sickle cell disease (SCD), continues to demonstrate sustained therapeutic benefits for as long as 20 months, or nearly two years. That’s according to new data covering 31 adults and adolescents with SCD who received risto-cel, formerly…
The gene-editing therapy Casgevy (exagamglogene autotemcel) safely and effectively prevents vaso-occlusive crises (VOCs) in children as young as 5 years old with severe sickle cell disease (SCD), according to preliminary data from a Phase 3 clinical trial. As of the latest follow-up, all children in the…
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