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Can serial cerebral MRIs predict the neuronopathic phenotype of MPS II?

Audrey A M VollebregtBerendine J EbbinkDimitris RizopoulosMaarten H LequinFemke K AarsenElsa G ShapiroAns T van der PloegJohanna M P van den Hout
Published in: Journal of inherited metabolic disease (2021)
In our cohort, brain atrophy showed a stronger correlation to a decline in CAS when compared to WMA. Atrophy-scores were higher in young neuronopathic patients than in non-neuronopathic patients and atrophy was an important early sign for the development of the neuronopathic phenotype, especially when observed jointly with white-matter abnormalities.
Keyphrases
  • end stage renal disease
  • white matter
  • newly diagnosed
  • ejection fraction
  • chronic kidney disease
  • prognostic factors
  • peritoneal dialysis
  • multiple sclerosis
  • crispr cas
  • blood brain barrier
  • middle aged