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Activity of daily living in mucopolysaccharidosis IVA patients: Evaluation of therapeutic efficacy.

Hui ChenShaukat KhanBetul CelikYasuyuki SuzukiYasuhiko AgoShunji Tomatsu
Published in: Molecular genetics & genomic medicine (2021)
Overall, this ADL questionnaire has demonstrated validation and reliability in assessing the MPS IVA patients and therapeutic efficacy.
Keyphrases
  • end stage renal disease
  • ejection fraction
  • chronic kidney disease
  • prognostic factors
  • peritoneal dialysis
  • patient reported outcomes
  • patient reported