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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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Detalles Bibliográficos
Autores principales: Staunton, Hannah, Trennery, Claire, Arbuckle, Rob, Guridi, Maitea, Zhuravleva, Elena, Furlong, Pat, Fischer, Ryan, Hall, Rebecca
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8314490/
https://www.ncbi.nlm.nih.gov/pubmed/34311756
http://dx.doi.org/10.1186/s12955-021-01813-w