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Home-Based Infusion of Alglucosidase Alfa Can Safely be Implemented in Adults with Late-Onset Pompe Disease: Lessons Learned from 18,380 Infusions.

Imke A M DittersHarmke A van KootenNadine A M E van der BeekJacqueline F HardonGamida IsmailovaEsther BrusseMichelle E KruijshaarAns T van der PloegJohanna M P van den HoutHidde H Huidekoper
Published in: BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy (2023)
Given the small numbers of IARs that occurred with the home infusions, of which only one was severe, we conclude that alglucosidase alfa can be administered safely in the home situation, provided the appropriate infrastructure is present.
Keyphrases
  • late onset
  • early onset
  • replacement therapy
  • healthcare
  • low dose