Home-based enzyme replacement therapy in children and adults with Pompe disease; a prospective study.

Imke A M DittersNadine A M E van der BeekEsther BrusseAns T van der PloegJohanna M P van den HoutHidde H Huidekoper

Published in: Orphanet journal of rare diseases (2023)

Our data demonstrate that home-based ERT in Pompe disease can be safely implemented as few, mostly mild, symptoms were reported during or after infusion. Insights from this study can be used as a base for implementing home-based ERT in other countries and to further optimize patient care, as unreported mild symptoms do not pose a health risk but may still be relevant to the patient.

Keyphrases

replacement therapy
health risk
smoking cessation
late onset
heavy metals
drinking water
case report
low dose
sleep quality
physical activity
early onset