{"database":"biostudies-literature","file_versions":[],"scores":null,"additional":{"omics_type":["Unknown"],"volume":["20(1)"],"submitter":["Toscano A"],"funding":["Sanofi"],"journal":["Orphanet journal of rare diseases"],"pagination":["639"],"full_dataset_link":["https://www.ebi.ac.uk/biostudies/studies/S-EPMC12752199"],"repository":["biostudies-literature"],"pubmed_title":["Long-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."],"pmcid":["PMC12752199"],"funding_grant_id":["Sanofi"],"pubmed_authors":["Siciliano G","Verrecchia E","Maggi L","Fiumara A","Taurisano R","Fischetto R","Toscano A","Barone R","Gasperini S","Musumeci O","Ravaglia S","Balzarini M","Parini R","Cianflone A","Cianci V","Brighina F","Sechi A","Scarpa M","Sacchini M","Crescimanno G"],"additional_accession":[]},"is_claimable":false,"name":"Long-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.","description":null,"dates":{"release":"2025-01-01T00:00:00Z","publication":"2025 Nov","modification":"2026-06-10T04:54:47.815Z","creation":"2026-06-10T03:07:27.73Z"},"accession":"S-EPMC12752199","cross_references":{"pubmed":["41272769"],"doi":["10.1186/s13023-025-04108-1"]}}