Development and validation of parent-reported gastrointestinal health scale in MECP2 duplication syndrome

Development and validation of parent-reported gastrointestinal health scale in MECP2 duplication syndrome

Abstract Background/aims We aimed to develop a validated patient-reported Gastrointestinal Health Scale (GHS) specific to MECP2 Duplication Syndrome (MDS) to be used in clinical trials. Methods MDS parents completed a Gastrointestinal Health Questionnaire (GHQ) to investigate the most relevant and i...

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Bibliographic Details
Main Authors: Davut Pehlivan, Sukru Aras, Daniel G. Glaze, Muharrem Ak, Bernhard Suter, Kathleen J. Motil
Format: Article
Language:English
Published: BMC 2024-02-01
Series:Orphanet Journal of Rare Diseases
Subjects:
MECP2 duplication syndrome
Parent-reported outcome
Gastrointestinal health scale
Validity
Reliability
Online Access:https://doi.org/10.1186/s13023-024-03022-2

Internet

https://doi.org/10.1186/s13023-024-03022-2

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