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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.
Toscano A, Musumeci O, Sacchini M, Ravaglia S, Siciliano G, Fiumara A, Verrecchia E, Maione M, Gentile J, Fischetto R, Crescimanno G, Taurisano R, Sechi A, Gasperini S, Cianci V, Maggi L, Parini R, Lupica A, Scarpa M. Toscano A, et al. Among authors: ravaglia s. Orphanet J Rare Dis. 2023 Oct 27;18(1):338. doi: 10.1186/s13023-023-02919-8. Orphanet J Rare Dis. 2023. PMID: 37891668 Free PMC article.
Ptosis as a feature of late-onset glycogenosis type II.
Ravaglia S, Repetto A, De Filippi P, Danesino C. Ravaglia S, et al. Neurology. 2007 Jul 3;69(1):116; author reply 116. doi: 10.1212/01.wnl.0000270101.95790.fb. Neurology. 2007. PMID: 17606892 No abstract available.
110 results