A high dose of pociredir, Fulcrum Therapeutics’ investigational oral therapy for sickle cell disease (SCD), was associated with increased fetal hemoglobin (HbF) levels and reduced markers of red blood cell destruction in early trial data. Those preliminary findings come from a Phase 1b clinical trial called PIONEER…
News
Risto-cel, Beam Therapeutics‘ single-dose, gene-edited cell therapy candidate for people with severe sickle cell disease (SCD), continues to demonstrate sustained therapeutic benefits for as long as 20 months, or nearly two years. That’s according to new data covering 31 adults and adolescents with SCD who received risto-cel, formerly…
The gene-editing therapy Casgevy (exagamglogene autotemcel) safely and effectively prevents vaso-occlusive crises (VOCs) in children as young as 5 years old with severe sickle cell disease (SCD), according to preliminary data from a Phase 3 clinical trial. As of the latest follow-up, all children in the…
Vascarta‘s VAS‑101, a skin gel containing curcumin — a component of the spice turmeric — was found in laboratory research to partially restore the health of mitochondria, the powerhouses of cells, in the hearts of a sickle cell disease (SCD) mouse model. “Treatment with VAS‑101 for 21 days…
Having a low household- or neighborhood-level socioeconomic status is not significantly associated with a higher risk of stroke in children with sickle cell disease (SCD). These findings are based on a secondary analysis of data from the observational DISPLACE study (NCT03621826), which was originally designed to identify barriers…
As part of a program to support children with sickle cell disease (SCD) and cancers, supplemental insurance company Aflac has released a new children’s book that aims to illustrate how empathy and compassion can help people, especially children, who are facing health problems. The book, titled “Beyond Words,” is…
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…
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