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 ToscanoOlimpia MusumeciMichele SacchiniSabrina RavagliaGabriele SicilianoAgata FiumaraElena VerrecchiaMelania MaioneJennifer GentileRita FischettoGrazia CrescimannoRoberta TaurisanoAnnalisa SechiSerena GasperiniVittoria CianciLorenzo MaggiRossella PariniAntonino LupicaMaurizio ScarpaPublished in: Orphanet journal of rare diseases (2023)
Evidence of favorable safety profile, improved treatment compliance and personal satisfaction validates the use of ERT with laronidase and alglucosidase alfa as a strong candidate for home therapy.
Keyphrases
- replacement therapy
- smoking cessation
- end stage renal disease
- coronavirus disease
- sars cov
- newly diagnosed
- ejection fraction
- chronic kidney disease
- healthcare
- high dose
- prognostic factors
- peritoneal dialysis
- type diabetes
- metabolic syndrome
- stem cells
- low dose
- late onset
- adipose tissue
- weight loss
- cell therapy
- glycemic control