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Article

Delandistrogene Moxeparvovec: First Approval.

Drugs - 1 Sept 2023

Hoy Sheridan M

Abstract excerpt

Delandistrogene moxeparvovec (delandistrogene moxeparvovec-rokl; ELEVIDYS®) is an adeno-associated virus (AAV) vector-based gene therapy designed to deliver a gene encoding a micro-dystrophin protein [i.e. a shortened (138 kDa) version of the dystrophin protein expressed in normal muscle cells (427 kDa)] to all muscles involved in the pathology of Duchenne muscular dystrophy (DMD). Developed by Sarepta...

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Identifiers

PMID: 37566211
DOI: 10.1007/s40265-023-01929-x

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