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Development of a Clinical Global Impression of Change (CGI-C) and a Caregiver Global Impression of Change (CaGI-C) measure for ambulant individuals with Duchenne muscular dystrophy

BACKGROUND: In clinical trials for rare diseases, such as Duchenne muscular dystrophy, clinical outcome assessments (COA) used to assess treatment benefit are often generic and may not be sensitive enough to detect change in specific patient populations. Thus, there is a need for disease specific CO...

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Bibliografiske detaljer
Udgivet i:Health Qual Life Outcomes
Main Authors: Staunton, Hannah, Trennery, Claire, Arbuckle, Rob, Guridi, Maitea, Zhuravleva, Elena, Furlong, Pat, Fischer, Ryan, Hall, Rebecca
Format: Artigo
Sprog:Inglês
Udgivet: BioMed Central 2021
Fag:
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC8314490/
https://ncbi.nlm.nih.gov/pubmed/34311756
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s12955-021-01813-w
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