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In this study of 22 patients around the world treated with the gene therapy: Three of the nine patients with the most severe form of the disease stopped transfusions The need for blood transfusions was reduced by 73% overall in those patients 12 of the 13 patients with a less serious form of the disease no longer needed transfusions The researchers also said there were no safety concerns after the treatment in the short term
Following these principles preserves peptide integrity and ensures consistent research results
Not currently listed on the WADA Prohibited List as of 2026
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