The Medical Letter TML n. 17/2012
Nr. 17 del 01/09/2012
TML n. 17/2012 – IN BREVE: Taliglucerasi per la malattia di Gaucher
La FDA ha approvato la taliglucerasi alfa (Elelyso-Pfizer/Protalix; non approvato dall’EMA), una forma ricombinante di glucocerebrosidasi, per il trattamento di pazienti adulti con malattia di Gaucher di tipo 1. In questi pazienti, un deficit genetico dell’enzima lisosomiale glucocerebrosidasi determina un accumulo di glucosilceramide nei lisosomi delle cellule reticoloendoteliali, principalmente nel fegato, nella milza e nel midollo osseo (1).
Questo è l'abstract dell'articolo. Per poter consultare l'intero numero o più numeri scegli il tipo di abbonamento che preferisci.
Se sei già abbonato accedi.
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