Sickle Cell Disease
Vivien Sheehan, MD, explained how a novel assay can guide personalized therapeutic approaches for sickle cell disease.
Rilzabrutinib significantly decreased spleen weight and reduced inflammation in mice with homozygous sickle cell genotype.
MIRCA is a device designed to assess the flexibility of red blood cells of patients with sickle cell disease.
Compared with placebo, crizanlizumab has superior safety and comparable efficacy in patients with sickle cell disease.
The study led by Paul George, MD, PhD, also highlights the importance of patient regimen adherence to secure these benefits.
The findings coincide with patient reports of experiencing increased vaso-occlusive episodes during menses.
The Foundation for Sickle Cell Disease Research will evaluate VAS-101 in a phase 1 clinical study.
The platform features incubation technology able to replicate cell microenvironments associated with SCD sickling.
Patients with follow-up of one month or longer saw hemolysis marker improvement and reported no vaso-occlusive crises.
In a phase III trial 90% of evaluable patients were free of severe vaso-occlusive crisis for at least 12 consecutive months.
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