The HRI signaling protein was identified as a key negative regulator of hemoglobin production in red blood cells, unraveling a potential new therapeutic target for sickle cell disease (SCD). Using the gene editing tool CRISPR-Cas9, HRI was found to block the production of fetal hemoglobin, which carries iron…
News
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…
A simple blood test and other measures could help halt the rise of sickle cell anemia in Africa and prevent fatal complications, especially among children, a World Heath Organization (WHO) official said this month. People living in Africa make up some 66 percent of the approximately 120 million people…
White blood cells were seen to produce molecules that decrease a specific type of hemoglobin — the protein in red blood cells that’s responsible for the transport of oxygen — in patients with sickle cell disease (SCD). These findings may help in the understanding of disease mechanisms, as well as resistance…
The Sickle Cell Foundation of Georgia (SCFG) has partnered with Biotech Research Laboratories and the Phoenix Tears Foundation to raise money for the first clinical study focused on the effectiveness of cannabis oil in sickle cell disease (SCD)-related pain management. The initiative is in honor of World…
Music therapy can help ease pain in adults with sickle cell disease (SCD), a pilot study showed. A single session of music improvisation with a music therapist helped significantly diminish the intensity of patients’ pain and uplift their moods, the study noted. The research, titled “…
Children with sickle cell disease who experience obstructive sleep apnea (OSA), a breathing disorder during sleep, are at risk of developing additional health problems, according to researchers. Their study, “Comorbid obstructive sleep apnea and increased risk for sickle cell disease morbidity,” was published in Sleep and…
Once-daily treatment with voxelotor, Global Blood Therapeutics’ lead therapy candidate, can effectively increase the amount of hemoglobin in about 58% of patients with sickle cell disease (SCD) in just 12 weeks, according to results from Part A of a Phase 3 trial. Supported by these positive results, the company…
Bluebird bio reported that its investigational gene therapy LentiGlobin showed promising results for severe sickle cell disease (SCD) patients in a Phase 1 clinical trial. Data from the ongoing HGB-206 Phase 1 multicenter study (NCT02140554) were revealed in an oral presentation, “Recent progress in gene therapy for severe…
The Sickle Cell Disease Association of America (SCDAA) is collaborating with Emmaus Life Sciences to increase patient education and awareness about the complications of sickle cell disease (SCD). The partners aim to improve national awareness so that information about the disease can reach broader audiences through online…
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