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

Background The Italian Medicines Agency (AIFA) demands precise information on benefit/risk profile of home-based enzyme replacement therapy (ERT) for the treatment of patients with Pompe disease and Mucopolysaccharidosis type I (MPS I). This passage is necessary to obtain the authorization for ERT h...
Ausführliche Beschreibung

Gespeichert in:
Autor*in:

Toscano, Antonio [verfasserIn]

Musumeci, Olimpia

Sacchini, Michele

Ravaglia, Sabrina

Siciliano, Gabriele

Fiumara, Agata

Verrecchia, Elena

Maione, Melania

Gentile, Jennifer

Fischetto, Rita

Crescimanno, Grazia

Taurisano, Roberta

Sechi, Annalisa

Gasperini, Serena

Cianci, Vittoria

Maggi, Lorenzo

Parini, Rossella

Lupica, Antonino

Scarpa, Maurizio

Format:

E-Artikel

Sprache:

Englisch

Erschienen:

2023

Schlagwörter:

Enzyme replacement therapy

Pompe disease

Home therapy

Mucopolysaccharidosis

Treatment adherence

Safety

Anmerkung:

© The Author(s) 2023

Übergeordnetes Werk:

Enthalten in: Orphanet journal of rare diseases - London : BioMed Central, 2006, 18(2023), 1 vom: 27. Okt.

Übergeordnetes Werk:

volume:18 ; year:2023 ; number:1 ; day:27 ; month:10

Links:

Volltext

DOI / URN:

10.1186/s13023-023-02919-8

Katalog-ID:

SPR053552938

Nicht das Richtige dabei?

Schreiben Sie uns!