News

A new oral treatment, mitapivat, for sickle cell disease (SCD) significantly boosted hemoglobin levels in patients over a year, achieving one of the primary goals of a Phase 3 clinical trial. In the RISE UP trial (NCT05031780), the medication helped patients 16 and older experience a key increase…

Researchers have identified a new method to reactivate fetal hemoglobin, without the need for gene-editing therapy, that has the potential to treat more people with sickle cell disease (SCD) at a lower cost and with fewer risks, as reported in a new preclinical study. While investigating the underlying mechanism…

Disc Medicine has launched a small clinical trial to test DISC-3405, its treatment candidate for reducing iron levels and easing iron overload, in people with sickle cell disease (SCD). The Phase 1b trial (NCT07187973) is expected to recruit 24 adults with SCD, who will receive the therapy at…

Treatment with hydroxyurea increases blood oxygen levels during sleep in children with sickle cell disease (SCD), but it does not meaningfully lessen sleep disturbances, a study reports. Compared with untreated SCD children, those given hydroxyurea (sold in the U.S. as Siklos and Xromi) had comparable sleep quality. Obstructive…

The first sickle cell disease (SCD) patient has been enrolled in a Phase 1b clinical trial evaluating iadademstat, Oryzon Genomics‘ investigational oral therapy designed to boost fetal hemoglobin (HbF). “SCD represents a major unmet medical need,” Carlos Buesa, PhD, Oryzon’s CEO, said in a company press release.

A Phase 1b clinical trial has exceeded the enrollment target for the group of adults with sickle cell disease (SCD) who will receive the highest dose of Fulcrum Therapeutics‘ experimental oral therapy pociredir. Previously reported data on the 12 mg dose group of the ongoing Phase 1b…

Disc Medicine says it’s planning to launch a clinical trial before the end of the year that will test DISC-3405, its treatment candidate for reducing high iron levels and easing iron overload, in people with sickle cell disease (SCD). “We’re … excited about the progress across our iron…

A blood test measuring a protein called placental growth factor (PlGF) may help identify women with sickle cell disease (SCD) who are at risk of developing early-onset preeclampsia, a new study shows. Preeclampsia is a disorder marked by high blood pressure and excess protein in the urine, which is…

The European Medicines Agency (EMA) has confirmed the continuing suspension of marketing authorization for Pfizer’s sickle cell disease (SCD) treatment Oxbryta (voxelotor) — withdrawn from the market last year due to safety concerns — following a committee’s recommendation that “concluded that the benefit-risk balance … is no longer favourable.” The EMA’s…

Using tocilizumab off-label in four young adults with sickle cell disease (SCD) who developed hyperhemolysis syndrome as a complication of a blood transfusion stopped the premature destruction of red blood cells, suggesting it may be safe and effective when other options fail. While tocilizumab is not approved for…