Marisa Wexler, MS,  senior science writer—

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…

The SECURE-SCD registry, which is collecting de-identified data on individuals with sickle cell disease (SCD) who develop COVID-19, will be expanding to include information on children and adults in Africa. The expansion was funded by a $100,000 grant from the Doris Duke Charitable Foundation (DDCF) awarded to…

A European Medicines Agency (EMA) committee has adopted a positive opinion on Novartis‘s Adakveo (crizanlizumab) for the prevention of vaso-occlusive crises (VOCs) in people with sickle cell disease (SCD). The EMA’s Committee for Medicinal Products for Human Use (CHMP) recommended the conditional approval of…

Blood stem cell transplant from unrelated donors is a feasible strategy in people with sickle cell disease (SCD), with better outcomes when blood markers are matched between donor and recipient, a study suggests. The study, “The role of HLA matching in unrelated donor hematopoietic stem cell…