Home-based enzyme replacement therapy in children and adults with Pompe disease; a prospective study.
Ditters IAM, van der Beek NAME, Brusse E, van der Ploeg AT, van den Hout JMP, Huidekoper HH
Orphanet journal of rare diseases, 2023 May 08
Abstract
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.