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Wedell-Neergaard, A

Crucially, the pivotal VALOR phase 3 trial did not meet its primary clinical endpoint (change in ALSFRS-R score at 28 weeks) (44)

628,629 The resultant mRNA is translated in the cytosol, 628,629 after which the mitochondrial targeting peptides facilitate the import of these proteins into mitochondria, achieving the allotopic expression of functional mtDNA genes to compensate for genetic defects