Tracking disease progression non‐invasively in Duchenne and Becker muscular dystrophies
Tracking disease progression non‐invasively in Duchenne and Becker muscular dystrophies
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Author / Creator
Spitali, Pietro , Hettne, Kristina , Tsonaka, Roula , Charrout, Mohammed , Bergen, Janneke , Koeks, Zaïda , Kan, Hermien E. , Hooijmans, Melissa T. , Roos, Andreas , Straub, Volker , Muntoni, Francesco , Al‐Khalili‐Szigyarto, Cristina , Koel‐Simmelink, Marleen J.A. , Teunissen, Charlotte E. , Lochmüller, Hanns , Niks, Erik H. and Aartsma‐Rus, Annemieke
Publisher
Germany: John Wiley & Sons, Inc
Journal title
Language
English
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Publication information
Publisher
Germany: John Wiley & Sons, Inc
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Scope and Contents
Contents
Background
Analysis of muscle biopsies allowed to characterize the pathophysiological changes of Duchenne and Becker muscular dystrophies (D/BMD) leading to the clinical phenotype. Muscle tissue is often investigated during interventional dose finding studies to show in situ proof of concept and pharmacodynamics effect of the tested drug. Less i...
Alternative Titles
Full title
Tracking disease progression non‐invasively in Duchenne and Becker muscular dystrophies
Authors, Artists and Contributors
Author / Creator
Hettne, Kristina
Tsonaka, Roula
Charrout, Mohammed
Bergen, Janneke
Koeks, Zaïda
Kan, Hermien E.
Hooijmans, Melissa T.
Roos, Andreas
Straub, Volker
Muntoni, Francesco
Al‐Khalili‐Szigyarto, Cristina
Koel‐Simmelink, Marleen J.A.
Teunissen, Charlotte E.
Lochmüller, Hanns
Niks, Erik H.
Aartsma‐Rus, Annemieke
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Primary Identifiers
Record Identifier
TN_cdi_doaj_primary_oai_doaj_org_article_0eb35a80d432451f8651ce90e2352016
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_0eb35a80d432451f8651ce90e2352016
Other Identifiers
ISSN
2190-5991,2190-6009
E-ISSN
2190-6009
DOI
10.1002/jcsm.12304