The Medical Letter TML n. 2/2017
Nr. 02 del 15/01/2017
TML n. 2/2017 – ETEPLIRSEN PER LA DISTROFIA MUSCOLARE DI DUCHENNE
L’eteplirsen (Exondys 51-Sarepta negli USA; in Europa il farmaco è oggetto di un piano d’indagine pediatrica [PIP]; non disponibile in commercio in Italia), un oligonucleotide antisenso, ha ricevuto l’approvazione mediante procedura accelerata della US Food and Drug Administration (FDA) per il trattamento della distrofia muscolare di Duchenne (DMD) nei pazienti che presentano una mutazione del gene della distrofina soggetta allo skipping (salto) dell’esone 51. È il primo farmaco a essere approvato per il trattamento della DMD.
Questo è l'abstract dell'articolo. Per poter consultare l'intero numero o più numeri scegli il tipo di abbonamento che preferisci.
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