STAT+: Fulcrum study suggests improved efficacy for higher dose sickle cell pill
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Fulcrum Therapeutics said Sunday that a higher dose of its experimental pill for sickle cell disease was more effective at inducing an alternative form of the oxygen-carrying molecule hemoglobin \u2014 boosting hopes it could one day provide a simple and effective treatment for the disease.\u00a0\u00a0<\/p>\n
Sickle cell is caused by a mutation in the hemoglobin gene, leading to misshapen blood cells that clog blood vessels and don\u2019t adequately deliver oxygen throughout the body. The pill, pociredir, is designed to induce fetal hemoglobin, an alternate form of the protein usually only active in the womb and infancy.<\/p>\n
In the early stage study presented<\/a> at the American Society of Hematology annual meeting, 12 participants who received 20mg daily pociredir saw their levels of fetal hemoglobin rise from 7.1% of the total hemoglobin in their blood to 16.9%.\u00a0<\/p>\n Continue to STAT+ to read the full story\u2026<\/a><\/p>\n<\/div>\n