African-Americans with sickle cell trait and sickle cell disease have a significantly faster decline in kidney function than their unaffected peers with normal hemoglobin, a study suggests. Men, people with diabetes, and those with good kidney function to start saw kidney function decline fastest, according to…
CTX001 safely and effectively increased the levels of fetal hemoglobin and prevented vaso-occlusive crises in the first severe sickle cell disease (SCD) patient receiving the therapy, according to preliminary data from a Phase 1/2 clinical trial. CTX001 is a CRISPR-based gene editing therapy developed by CRISPR Therapeutics and Vertex…
Treatment with Endari (L-glutamine) leads to fewer pain crises and hospitalizations in patients with sickle cell disease, according to final results of a Phase 3 clinical trial. These results were published in the study, “A Phase 3 Trial of l-Glutamine in Sickle Cell Disease,” in the…
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