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Start, switch and stop (triple-S) criteria for enzyme replacement therapy of late-onset Pompe disease: European Pompe Consortium recommendation update 2024.

Benedikt SchoserNadine A M E van der BeekAlexander BroomfieldEsther BrusseJordi Díaz-ManeraAndreas HahnThomas HundsbergerCornelia KornblumMichelle KruijshaarPascal LaforêtEugen MengelTiziana MonginiDavid OrlikowskiGiancarlo ParentiW W M Pim PijnappelMark RobertsThomas SchererAntonio ToscanoJohn VissingJohanna M P van den HoutPieter A van DoornStephan WenningerAns T van der Ploeg
Published in: European journal of neurology (2024)
The triple-S criteria on ERT start, switch and stop include muscle magnetic resonance imaging as a supportive finding and the potential option of home infusion therapy. Six-monthly long-term monitoring of muscle function is highly recommended to cover insights into the patient's trajectory under ERT.
Keyphrases
  • late onset
  • replacement therapy
  • magnetic resonance imaging
  • early onset
  • skeletal muscle
  • smoking cessation
  • case report
  • healthcare
  • computed tomography
  • stem cells
  • contrast enhanced
  • risk assessment
  • magnetic resonance