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10.1126/scisignal.adh1178 16 CaoY.ChenJ.XiaoJ.HongY.XuK.ZhuY

STX209 (arbaclofen) for autism spectrum disorders: an 8-week open-label study

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798 This mechanism restores mitochondrial targeting sequence pre-processing and alleviates feedback inhibition of mitochondrial processing peptidase activity in fibroblasts from PITRM1-deficient patients, improving Frataxin maturation and mitochondrial function