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With the development of highly sensitive techniques based on mass spectrometry to determine acylcarnitine profiles in blood, urine or dried blood spots (DBS), a fast screening method for lcFAOD has become available [34, 35]

However, in practice, investigators could only introduce a small amount of stratification and could not balance all important variables simultaneously ( Propensity score matching (PSM) was defined as the conditional probability of a subject being assigned to the treatment group given the observed covariates

Revisin actualizada de los aspectos clnicos, diagnsticos y teraputicos de la enfermedad de Niemann-Pick C

doi:10.1002/ptr.3294