Development of Exon Skipping Therapies for Duchenne Muscular Dystrophy: A Critical Review and a Perspective on the Outstanding Issues

Author(s):: Aartsma-Rus, A.; Straub, V.; Hemmings, R.; Haas, M.; Schlosser-Weber, G.; Stoyanova-Beninska, V.; Mercuri, E.; Muntoni, F.; Sepodes, B.; Vroom, E.; Balabanov, P.

Published: 2017
Publication type: Article / Letter to editor

exon skippingoligonucleotidesregulatory approval

Date issued: 2017-10-31
Language: English
Is part of: Nucleic Acid Therapeutics (2017), Vol. 27, no. 5, p. 251-259
hdl: 1887/94902
doi: 10.1089/nat.2017.0682