Marisa Wexler, MS, senior science writer —

LentiGlobin, Bluebird Bio’s investigational gene therapy for sickle cell disease (SCD), is unlikely to be the cause of a blood cancer that arose in one of the patients who received the therapy in an early clinical trial, according to new analyses. In February, Bluebird paused two clinical…

Red blood cells in people with sickle cell disease (SCD) flow through blood vessels in a manner that damages the walls, which may explain blood vessel problems and increased inflammation in SCD, new research indicates. The study, “Flow-induced segregation and dynamics of red blood cells in sickle cell…

The European Medicines Agency (EMA) is reviewing an application from Global Blood Therapeutics (GBT) that seeks full marketing approval for the oral therapy Oxbryta (voxelotor) for people with sickle cell disease (SCD), ages 12 and up. Oxbryta would be the first treatment targeting the root cause of…

FT-4202, a potential disease-modifying oral treatment for sickle cell disease (SCD), is well-tolerated in people with SCD and induces improvements in several red blood cell parameters, early clinical trial data indicate. The results observed in this initial group of patients support the efficacy of Forma Therapeutics‘ investigational medication, as…

A team of researchers led by UNC Project-Malawi has been given a one-year, $500,000 grant to build clinical and research infrastructure that will improve diagnosis and treatment for children living with sickle cell disease (SCD) in Malawi. Malawi is a country located in sub-Saharan Africa. Each year…

The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of free virtual workshops in December, with the aim of empowering leaders to start non-profit organizations and research programs to help people with rare diseases.  “The RareLaunch program is…

A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like the option of continuing its use in future appointments, a series of surveys found. The surveys were conducted by the National Organization for Rare Disorders (NORD) and involved more than 800…

FTX-6058, an investigational treatment for sickle cell disease (SCD) and beta-thalassemia being developed by Fulcrum Therapeutics, showed efficacy at raising the levels of fetal hemoglobin in cellular and animal models of the diseases, according to new preclinical data announced by the company. Fulcrum also stated…

Medications that activate the protein soluble guanylyl cyclase (sGC) may have promise in the treatment of sickle cell disease (SCD), a new study in cells and animal models indicates. The study, “Beneficial Effects of Soluble Guanylyl Cyclase Stimulation and Activation in Sickle Cell Disease Are…

Using a newly developed assay, researchers identified seven U.S. Food and Drug Administration (FDA)-approved medications that may be effective, with minimal safety issues, in treating sickle cell disease (SCD). Their findings were reported in the Journal of Clinical Medicine, in the study, “Large-Scale Drug Screen Identifies…