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The problem
Intervention: High-dose methionine supplementation started at median age of 6 months
Who it was tested with: 16 children aged 14 months to 7.3 years with MARS1-related PAP.
The same group is measured before the intervention and again afterwards, with a baseline and an endline and sometimes a midline. Change is visible, but other causes cannot be ruled out.
"Intellectual functioning was assessed using the Wechsler Preschool and Primary Scale of Intelligence, Fourth Edition (WPPSI-IV) when age-appropriate, and adaptive behavior was evaluated via the Vineland Adaptive Behavior Scales, Second Edition (VABS-II) through parent interviews." The study used validated neuropsychological measures to characterize neurodevelopmental outcomes in children receiving methionine, finding significant impairments despite treatment, indicating the intervention does not prevent the neurodevelopmental effects of the disease.
Small sample size with only 8 of 16 children able to complete IQ testing; no comparison group to determine if impairments are disease-specific; no baseline measurement before methionine initiation to establish effect; cross-sectional design limits causal inference about methionine's role in developmental outcomes
Across countries and designs
1 study · 1 country
