Development of Exon Skipping Therapies for Duchenne Muscular Dystrophy: A Critical Review and a Perspective on the Outstanding Issues
Aartsma-Rus, Annemieke, Straub, Volker, Hemmings, Robert, Haas, Manuel, Schlosser-Weber, Gabriele, Stoyanova-Beninska, Violeta, Mercuri, Eugenio, Muntoni, Francesco, Sepodes, Bruno, Vroom, Elizabeth, Balabanov, Pavel
Published in Nucleic acid therapeutics (01.10.2017)
Published in Nucleic acid therapeutics (01.10.2017)
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Journal Article
Development of Exon Skipping Therapies for Duchenne Muscular Dystrophy: A Critical Review and a Perspective on the Outstanding Issues
Aartsma-Rus, Annemieke, Straub, Volker, Hemmings, Robert, Haas, Manuel, Schlosser-Weber, Gabriele, Stoyanova-Beninska, Violeta, Mercuri, Eugenio, Muntoni, Francesco, Sepodes, Bruno, Vroom, Elizabeth, Balabanov, Pavel
Published in Nucleic acid therapeutics (01.10.2017)
Published in Nucleic acid therapeutics (01.10.2017)
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Journal Article
European Medicines Agency review of ataluren for the treatment of ambulant patients aged 5 years and older with Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene
Haas, Manuel, Vlcek, Viktor, Balabanov, Pavel, Salmonson, Tomas, Bakchine, Serge, Markey, Greg, Weise, Martina, Schlosser-Weber, Gabriele, Brohmann, Henning, Yerro, Concepcion Prieto, Mendizabal, Macarena Rodriguez, Stoyanova-Beninska, Violeta, Hillege, Hans L
Published in Neuromuscular disorders : NMD (01.01.2015)
Published in Neuromuscular disorders : NMD (01.01.2015)
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Journal Article