Oxbryta (voxelotor), the first treatment for sickle cell disease that addresses a root cause, will continue to be tested in a post-approval confirmatory study opening soon in children as young as age 2, according to its developer, Global Blood Therapeutics (GBT). The new study, called HOPE-KIDS 2, is…
Oxbryta (voxelotor), a first sickle cell treatment that works to stop the sickling and destruction of red blood cells that mark this disease, has been given accelerated approval by the U.S. Food and Drug Administration (FDA) for patients ages 12 and older. Specifically, Oxbryta — an oral,…
Droplets packed with hemoglobin may briefly form tiny clusters inside red blood cells that ultimately deform their shape, and cause the pain crises that mark sickle cell disease (SCD), a study reports. The research, “A mechanism for reversible mesoscopic aggregation in liquid solutions,” appeared in the journal …
Adakveo (crizanlizumab) is an “important addition” to the care of sickle cell disease, a safe and effective therapy that works against painful vaso-occlusive crises (VOCs) in SCD patients and is likely do so over the long term, said the doctor who led the clinical trial that…
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…
Universal screening to identify socioeconomic needs of children and families affected by sickle cell disease can help to address their specific difficulties, and ultimately improve the healthcare of those with the disease, a study suggests. The study, “Universal screening for social determinants of health in pediatric sickle…
A first targeted treatment for sickle cell disease (SCD) — Adakveo (crizanlizumab), by Novartis, that works to lower the frequency of the painful vaso-occlusive crises (VOCs) common in this disease — has been approved by the U.S. Food and Drug Administration (FDA). Adakveo…
Rare disease-themed videos glowed on a large screen before an audience of people in wheelchairs, with crutches, and bearing oxygen tanks this Nov. 9 and 10 in San Francisco. Disorder: The Rare Disease Film Festival strives to eventually host a film about every one of the nearly 7,000 rare…
Scientists have found a way to increase the effectiveness of the gene-editing tool CRISPR/Cas9 that one day may be used to treat people with sickle cell disease (SCD) and HIV. The findings were reported in the study, “Improved Cas9 activity by specific modifications of the tracrRNA,”…
A technique called zinc finger nuclease (ZFN) gene editing technology can be used to modify immature red blood cells — called precursor cells — to boost the production of fetal hemoglobin and help ensure red blood cells maintain a normal shape in people with sickle cell disease (SCD),…
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