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),…
Treatment with delta-aminolevulinate (ALA), a precursor of heme — the part of hemoglobin that carries oxygen — could be a future therapy option for people with sickle cell anemia and β-thalassemia, a recent study suggests. The study, “δ-Aminolevulinate induces fetal hemoglobin expression by enhancing…
White blood cells, especially monocytes, may underlie the breathing difficulties affecting children with sickle cell disease (SCD), a small pilot study suggests. Asthma is estimated to impact from 17% to 28% of all children with SCD, its researchers noted, and obstructive lung disease — which affects exhaling — is…
The National Institutes of Health (NIH) and the Bill & Melinda Gates Foundation will each invest $100 million over the next four years to speed the development of affordable gene therapies for sickle cell disease (SCD) and the human immunodeficiency virus (HIV) on a global scale.
Enhanced parenting skills, including attentive listening and engaging in conversations, may help improve cognitive functioning in children with sickle cell disease (SCD), a study suggests. Parent stress was related to lower parenting responsiveness, which also may lead to lower cognitive function in children, the researchers said. The study, “…
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