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progressive dilated cardiomyopathy in a 24 year old man a rare manifestation of propionic acidemia
Original title: “Progressive Dilated Cardiomyopathy in a 24-Year-Old Man: A Rare Manifestation of Propionic Acidemia.”
propionic acidemia in mexico clinical and genotypic spectrum
Original title: “Propionic acidemia in Mexico: Clinical and genotypic spectrum.”
from n of 1 to versatility in propionic acidemia antisense oligonucleotide mediated skipping of a constitutive pcca pseudoexon
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.”