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Quality of life in Barth syndrome.
Ther Adv Rare Dis. 2022 Jun 11;3:26330040221093743. doi: 10.1177/26330040221093743. eCollection 2022 Jan-Dec.
Ther Adv Rare Dis. 2022.
PMID: 37180415
Free PMC article.
Initial Psychometric Evaluation of the Barth Syndrome Symptom Assessment (BTHS-SA) for Adolescents and Adults in a Phase 2 Clinical Study.
Gwaltney C, Shields A, Love E, Ollis S, Stokes J, Mazar I, Arenson E, Aiudi A, Wirth RJ, Houts C.
Gwaltney C, et al.
Orphanet J Rare Dis. 2025 Apr 25;20(1):199. doi: 10.1186/s13023-025-03693-5.
Orphanet J Rare Dis. 2025.
PMID: 40281531
Free PMC article.
Clinical Trial.
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