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propionic acidemia in mexico clinical and genotypic spectrum
Original title: “Propionic acidemia in Mexico: Clinical and genotypic spectrum.”
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Original title: “From N-of-1 to versatility in propionic acidemia: Antisense oligonucleotide-mediated skipping of a constitutive PCCA pseudoexon.”
characterizing the frequency of clinical events and assessing biomarkers in propionic acidemia a natural history study
Original title: “Characterizing the frequency of clinical events and assessing biomarkers in propionic acidemia: a natural history study.”