FDA Approves Eteplirsen for Duchenne Muscular Dystrophy: The Next Chapter in the Eteplirsen Saga
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| Vydáno v: | Nucleic acid therapeutics Ročník 27; číslo 1; s. 1 - 3 |
|---|---|
| Hlavní autoři: | , |
| Médium: | Journal Article |
| Jazyk: | angličtina |
| Vydáno: |
Los Angeles, CA
Mary Ann Liebert, Inc
01.02.2017
SAGE Publications |
| Témata: | |
| ISSN: | 2159-3337, 2159-3345, 2159-3345 |
| On-line přístup: | Získat plný text |
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| Author | Krieg, Arthur M. Aartsma-Rus, Annemieke |
|---|---|
| Author_xml | – sequence: 1 givenname: Annemieke surname: Aartsma-Rus fullname: Aartsma-Rus, Annemieke organization: 1Leiden University Medical Center, Leiden, the Netherlands – sequence: 2 givenname: Arthur M. surname: Krieg fullname: Krieg, Arthur M. organization: 2Checkmate Pharmaceuticals, Cambridge, Massachusetts |
| BackLink | https://www.ncbi.nlm.nih.gov/pubmed/27929755$$D View this record in MEDLINE/PubMed |
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| References | Mendell, Rodino-Klapac, Sahenk, Roush, Bird, Lowes, Alfano, Gomez, Lewis 2013; 74 Straub, Balabanov, Bushby, Ensini, Goemans, De Luca, Pereda, Hemmings, Campion 2016; 15 Goemans, Tulinius, van den Hauwe, Kroksmark, Buyse, Wilson, van Deutekom, de Kimpe, Lourbakos, Campion 2016; 11 Mendell, Goemans, Lowes, Alfano, Berry, Shao, Kaye, Mercuri 2016; 79 B2 B4 B7 B14 26573217 - Ann Neurol. 2016 Feb;79(2):257-71 23907995 - Ann Neurol. 2013 Nov;74(5):637-47 27302365 - Lancet Neurol. 2016 Jul;15(8):882-90 27588424 - PLoS One. 2016 Sep 02;11(9):e0161955 |
| References_xml | – volume: 79 start-page: 257 year: 2016 end-page: 271 article-title: Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy publication-title: Ann Neurol – volume: 74 start-page: 637 year: 2013 end-page: 647 article-title: Eteplirsen for the treatment of Duchenne muscular dystrophy publication-title: Ann Neurol – volume: 15 start-page: 882 year: 2016 end-page: 890 article-title: Stakeholder cooperation to overcome challenges in orphan medicine development: the example of Duchenne muscular dystrophy publication-title: Lancet Neurol – volume: 11 start-page: e0161955 year: 2016 article-title: Long-term efficacy, safety, and pharmacokinetics of drisapersen in Duchenne muscular dystrophy: results from an open-label extension study publication-title: PLoS One – ident: B7 doi: 10.1002/ana.24555 – ident: B2 doi: 10.1371/journal.pone.0161955 – ident: B4 doi: 10.1002/ana.23982 – ident: B14 doi: 10.1016/S1474-4422(16)30035-7 – reference: 23907995 - Ann Neurol. 2013 Nov;74(5):637-47 – reference: 27588424 - PLoS One. 2016 Sep 02;11(9):e0161955 – reference: 27302365 - Lancet Neurol. 2016 Jul;15(8):882-90 – reference: 26573217 - Ann Neurol. 2016 Feb;79(2):257-71 |
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| SubjectTerms | Adolescent Child Clinical Trials as Topic Dose-Response Relationship, Drug Drug Approval Dystrophin - genetics Exons Humans Issues in Development Morpholinos - therapeutic use Muscular Dystrophy, Duchenne - drug therapy Muscular Dystrophy, Duchenne - economics Oligonucleotides - therapeutic use Time Factors Treatment Outcome United States United States Food and Drug Administration Walk Test Young Adult |
| Title | FDA Approves Eteplirsen for Duchenne Muscular Dystrophy: The Next Chapter in the Eteplirsen Saga |
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