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Home-based enzyme replacement therapy in children and adults with Pompe disease

by informer
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Pompe disease is a lysosomal storage disease treated with life-long enzyme replacement therapy (ERT). Home-based ERT has been provided in the Netherlands since 2008 because it diminishes the burden of treatment, increases patient flexibility and autonomy, and is thus a more patient-centred approach to ERT.

In this report all Dutch Pompe patients receiving alglucosidase alfa infusions at home are approached to participate in a questionnaire to validate the safety of home-based ERT. Prospective data on symptoms occurring during or within 48 h after infusion and retrospective data on infusion associated reactions (IARs) in the last three months are collected four times during one year.

The data in this study demonstrate that home-based ERT in Pompe disease can be safely implemented as few, mostly mild, symptoms are 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. Read the full article here.

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