Steve Bryson, PhD, science writer —

Nearly 3 in every 4 people with sickle cell disease (SCD) in the U.S. are not using an approved, disease-modifying therapy (DMT), according to a nationwide claims analysis. These patients tend to be older and to have relatively milder disease — the lowest prevalence of vaso-occlusive crises (VOCs)…

Treatment with hydroxyurea reduced the incidence of severe or invasive infections by 60% among children in Uganda with sickle cell anemia, the most common and usually the most severe form of sickle cell disease, a recent study reports. “Our investigation provides powerful…

Ferriprox (deferiprone) showed comparable efficacy to Desferal (deferoxamine mesylate) in reducing blood transfusion-induced iron overload in children and adolescents with sickle cell disease (SCD) or other anemias, a recent study reported. Ferriprox is an oral treatment, while Desferal is administered via a subcutaneous (under-the-skin) infusion. As such, Ferriprox is…

A small proof-of-concept clinical trial evaluating motixafortide for stem cell mobilization — regimens essential for gene therapies — in sickle cell disease (SCD) has dosed its first patient. Stem cell mobilization regimens currently available can cause serious side effects in SCD patients. This Phase 1 study (NCT05618301),…

Throughout 2023, Sickle Cell Disease News brought readers coverage of the latest clinical research and scientific breakthroughs related to sickle cell disease (SCD). Here are the year’s top 10 most-read articles, each with a brief description. We hope to remain a dependable resource for the SCD community in…

Rates of life-threatening infections by pneumococcal bacteria have significantly declined, by about 80%, among children with sickle cell disease (SCD) since the introduction of pneumococcal conjugate vaccines 20 years ago. That’s according to a study analyzing more than two decades of healthcare data from young SCD patients in metropolitan…

Treatment with the investigational gene-editing therapy OTQ923 led to the sustained production of fetal hemoglobin and prevented vaso-occlusive crises (VOCs) in three adults with severe sickle cell disease (SCD), according to data from a Phase 1 study. The treatment involved collecting red blood cell precursors, called hematopoietic stem…

Preventive red blood cell transfusions may protect against complications associated with pregnancy in women with sickle cell disease (SCD), a 13-year study has found. Findings indicate that the rates of premature births, as well as SCD complications, such as vaso-occlusive crises (VOCs) and acute chest syndrome (ACS), are…

Lobe Sciences has partnered with Pentec Health to distribute Altemia, an emulsified formulation of docosahexaenoic acid (DHA), a type of omega-3 fat, for sickle cell disease (SCD) patients in the U.S. and its territories. As part of the agreement, Pentec, which provides clinical nutrition and specialty care…

The investigational oral treatment FTX-6058 effectively raised the level of fetal hemoglobin in a sickle cell disease (SCD) patient to 24.9% in a clinical trial that’s now been placed on hold by regulators. According to the therapy’s developer, Fulcrum Therapeutics, these initial data had been obtained but not…