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Prof Douglas Higgs, also from Oxford University, pointed out that not all the patients had become free of transfusions and there was still little known about the long-term effects of manipulating the genome of stem cells in this way

The compound appears well-tolerated in animals at doses well above those producing metabolic effects

A 23-week randomized clinical trial found that participants taking 1 mg of AOD-9604 daily lost an average of 6.2 pounds compared to 1.8 pounds in the placebo group

The model identifies pathways responsible for the effect of the double and triple drug combinations, versus the effect of the addition of each single agent at its IC50 concentration