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Competing Interests: Declaration of Competing Interest RM, MH, and YC report grants, non-financial support, personal fees and other suppot from Zogenix/UCB. In addition, RM and MH have a patent “Deoxynucleoside therapy for diseases caused by unbalanced nucleotide pools including mitochondrial DNA depletion syndromes” (PCT/US16/038110) with royalties paid by Zogenix/UCB; MH reports grants and other support from Entrada Therapeutics. RM and YC have a patent “Treatment of mitochondrial diseases” (PCT/EP2016/062636) with royalties paid by Zogenix/UCB.These relationships for MH are de minimus for Columbia University Irving Medical Center and for RM and YC are de minimus for Vall d'Hebron Research Institute and CIBERER.