Hydroxyurea, blood transfusions, and donor blood stem cell transplants can significantly lower the risk of stroke in children and adolescents with sickle cell anemia (SCA), the most common and severe form of sickle cell disease. That’s according to a systematic review and meta-analysis of 40 studies that also highlighted…
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Big Nova has made a $3 million donation to support NMDP’s efforts to help more patients, including those with sickle cell disease (SCD), access blood stem cell transplants. The philanthropic gift helped NMDP exceed its $100 million fundraising goal, a milestone the organization said will support its…
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…
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…
Recent Posts
- Standard sickle cell treatments slash stroke risk in children: Review
- Is sickle cell disease considered a disability?
- Tips for enjoying the most wonderful time of the year with sickle cell
- Big Nova donates $3M to expand access to stem cell transplants
- Investigational SCD therapy shows stronger results at higher dose
- Risto-cel showing lasting benefits for people with severe SCD in trial
- The price of living with a physical disability in a non-inclusive society
- Casgevy safely prevents sickle cell crises in children: Trial data
- Curcumin skin gel shown to improve heart health in SCD mice
- Socioeconomic status not linked to stroke risk in children with SCD