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This made SS-31 the first drug ever authorized by the FDA specifically for Barth syndrome, and one of the first mitochondrially-targeted drugs to receive FDA approval for any indication.[3] Compared to MOTS-c — another mitochondria-targeted research compound — SS-31 acts upstream at the level of cardiolipin and the electron transport chain itself, rather than via nuclear gene regulation.[1] Discovery and design rationale SS-31 was identified by Szeto and Schiller during structure-activity dissection of opioid-receptor agonists derived from [Dmt¹]DALDA (SS-02) , a synthetic δ-opioid peptide analog originally explored as an analgesic scaffold