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The same parent completed the PedsQL at each visit and children five years of age and older completed the age-appropriate PedsQL

The choices of inclusion criteria, dose, duration, and outcome variables were necessarily based on our best hypotheses given the lack of previous studies with VPA in children with SMA, and limited experience in rigorous clinical studies in this population

The results have important implications for the model built to represent this biotransformation, underlining that proper expression of the central metabolism, affecting balanced cofactor levels (acetyl-CoA/CoA in this case), influences secondary metabolism pathways [26]

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it also often indicates the end of life