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Safety Outcomes and Patients' Preferences for Home-Based Intravenous Enzyme Replacement Therapy (ERT) in Pompe Disease and Mucopolysaccharidosis Type I (MPS I) Disorder: COVID-19 and Beyond.

Antonio Toscano,Olimpia Musumeci,Michele Sacchini,Sabrina Ravaglia,Gabriele Siciliano,Agata Fiumara,Elena Verrecchia, Melania Maione, Jennifer Gentile,Rita Fischetto,Grazia Crescimanno,Roberta Taurisano,Annalisa Sechi,Serena Gasperini,Vittoria Cianci,Lorenzo Maggi,Rossella Parini,Antonino Lupica,Maurizio Scarpa

ORPHANET JOURNAL OF RARE DISEASES（2023）

Cited 0|Views18
Key words
Enzyme replacement therapy,Pompe disease,Home therapy,Mucopolysaccharidosis,Treatment adherence,Safety
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