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…
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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…
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…
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