Long-Term Safety and Efficacy Data of Golodirsen in Ambulatory Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A First-in-human, Multicenter, Two-Part, Open-Label, Phase 1/2 Trial.
| Autor: | Servais, Laurent, Mercuri, Eugenio, Straub, Volker, Guglieri, Michela, Seferian, Andreea M., Scoto, Mariacristina, Leone, Daniela, Koenig, Erica, Khan, Navid, Dugar, Ashish, Wang, Xiaodong, Han, Baoguang, Wang, Dan, Muntoni, Francesco, Brusa, Chiara, Antonaci, Laura, Brogna, Claudia, Merli, Laura, Monforte, Mauro, Norcia, Giulia |
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| Zdroj: | Nucleic Acid Therapeutics; Feb2022, Vol. 32 Issue 1, p29-39, 11p |
| Databáze: | Complementary Index |
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