biostudies-literatureUnknown20(1)Toscano ASanofiOrphanet journal of rare diseases639https://www.ebi.ac.uk/biostudies/studies/S-EPMC12752199biostudies-literatureLong-term safety outcomes and patient preferences for home-based intravenous enzyme replacement therapy (ERT) in Pompe disease and Mucopolysaccharidosis Type I (MPS-I): final results of two-year observation.PMC12752199SanofiSiciliano GVerrecchia EMaggi LFiumara ATaurisano RFischetto RToscano ABarone RGasperini SMusumeci ORavaglia SBalzarini MParini RCianflone ACianci VBrighina FSechi AScarpa MSacchini MCrescimanno GfalseLong-term safety outcomes and patient preferences for home-based intravenous enzyme replacement therapy (ERT) in Pompe disease and Mucopolysaccharidosis Type I (MPS-I): final results of two-year observation.2025-01-01T00:00:00Z2025 Nov2026-06-10T04:54:47.815Z2026-06-10T03:07:27.73ZS-EPMC127521994127276910.1186/s13023-025-04108-1