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

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

Abstract 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 spe...

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Bibliographic Details
Main Authors: Hannah Staunton, Claire Trennery, Rob Arbuckle, Maitea Guridi, Elena Zhuravleva, Pat Furlong, Ryan Fischer, Rebecca Hall
Format: Article
Language:English
Published: BMC 2021-07-01
Series:Health and Quality of Life Outcomes
Subjects:
Duchenne muscular dystrophy
Quality of life
Children
Adolescent
Clinical outcome assessment
Online Access:https://doi.org/10.1186/s12955-021-01813-w

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