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

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 treatment...

Descripción completa

Detalles Bibliográficos
Autores principales: Gwaltney, Chad, Stokes, Jonathan, Aiudi, Anthony, Mazar, Iyar, Ollis, Sarah, Love, Emily, Shields, Alan
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2021
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8190857/
https://www.ncbi.nlm.nih.gov/pubmed/34108006
http://dx.doi.org/10.1186/s13023-021-01897-z

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8190857/
https://www.ncbi.nlm.nih.gov/pubmed/34108006
http://dx.doi.org/10.1186/s13023-021-01897-z

Ejemplares similares