Cost-effectiveness of enzyme replacement therapy with alglucosidase alfa in adult patients with Pompe disease

BACKGROUND: Pompe disease is a rare, progressive, metabolic disease, and the first treatable inheritable muscle disorder. Enzyme replacement therapy (ERT) with alglucosidase alfa is disease specific and the only medicinal product authorized for the treatment of Pompe disease. Costs of ERT are very h...

Descrizione completa

Salvato in:
Dettagli Bibliografici
Pubblicato in:Orphanet J Rare Dis
Autori principali: Kanters, Tim A., van der Ploeg, Ans T., Kruijshaar, Michelle E., Rizopoulos, Dimitris, Redekop, W. Ken, Rutten-van Mӧlken, Maureen P. M. H., Hakkaart-van Roijen, Leona
Natura: Artigo
Lingua:Inglês
Pubblicazione: BioMed Central 2017
Soggetti:
Research
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC5729274/
https://ncbi.nlm.nih.gov/pubmed/29237491
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-017-0731-0
Tags: Aggiungi Tag
Nessun Tag, puoi essere il primo ad aggiungerne! !

Documenti analoghi