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continuing suspension of marketing authorization for Pfizer’s sickle cell disease (SCD) treatment Oxbryta (voxelotor) — withdrawn from the market last year due to safety concerns — following a committee’s recommendation that “concluded that the benefit-risk balance … is no longer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…
New research shows that Black men with sickle cell disease (SCD) become biological parents at a lower rate than similarly aged Black men in the U.S. general population. The analysis found about 25% fewer Black men enrolled in the Sickle Cell Disease Implementation Consortium (SCDIC) registry were parents compared…
People with sickle cell disease (SCD) seeking emergency care for vaso-occlusive crises (VOCs) are often not triaged appropriately, leading to unnecessarily long waits for pain relief. A recent study finds that assigning the correct Emergency Severity Index score, a tool used in emergency departments to prioritize care based…
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