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...

Popoln opis

Bibliografske podrobnosti

Principais autores:	Chad Gwaltney, Jonathan Stokes, Anthony Aiudi, Iyar Mazar, Sarah Ollis, Emily Love, Alan Shields
Format:	Artigo
Jezik:	Inglês
Izdano:	BMC 2021-06-01
Serija:	Orphanet Journal of Rare Diseases
Teme:	Content validity Instrument development Patient-reported outcomes Barth Syndrome Barth Syndrome Symptom Assessment
Online dostop:	https://doi.org/10.1186/s13023-021-01897-z
Oznake:	Označite Brez oznak, prvi označite!