Development and content validity of the Barth Syndrome Symptom Assessment (BTHS-SA) for adolescents and adults

Development and content validity of the Barth Syndrome Symptom Assessment (BTHS-SA) for adolescents and adults

Abstract Background Barth Syndrome (BTHS) is a rare genetic disorder that presents as a complex of debilitating symptoms and reduced life expectancy. Well-developed, BTHS-specific assessments measuring primary signs and symptoms of BTHS are not currently available, making it difficult to evaluate tr...

Full description

Bibliographic Details
Main Authors: Chad Gwaltney, Jonathan Stokes, Anthony Aiudi, Iyar Mazar, Sarah Ollis, Emily Love, Alan Shields
Format: Article
Language:English
Published: BMC 2021-06-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Content validity
Instrument development
Patient-reported outcomes
Barth Syndrome
Barth Syndrome Symptom Assessment
Online Access:https://doi.org/10.1186/s13023-021-01897-z

Internet

https://doi.org/10.1186/s13023-021-01897-z

Similar Items