The Medical Letter TML n. 17 / 2024
Nr. 17 del 19/08/2024
TML n. 17 / 2024 Indicazione ampliata per Elevidys
La terapia genica basata sul virus adenoassociato (AAV) delandistrogene moxeparvovec (Elevidys - Sarepta in USA; non disponibile in commercio in italia) ha ricevuto l'approvazione accelerata dalla US Food and Drug Administration (FDA) nel 2023 per il trattamento ambulatoriale di bambini di quattro-cinque anni affetti da distrofia muscolare di Duchenne (DMD) che presentano una mutazione confermata nel gene DMD. (1) Ora ha ricevuto l'approvazione completa per l'uso ambulatoriale in pazienti di età pari o superiore ai quattro anni e l'approvazione accelerata per l'uso in pazienti non ambulatoriali con DMD.
MECCANISMO D'AZIONE – Il delandistrogene moxeparvovec è una terapia non replicante, basata su un vettore AAV ricombinante che rilascia un transgene codificante la proteina microdistrofina, una proteina che contiene domini selezionati della distrofina espressa nelle cellule muscolari normali.
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