Publications by authors named "T R Butt"
Purpose: The multisystem clinical manifestations and complications of achondroplasia, the most common form of disproportionate short stature, can cause functional impairment and psychosocial burden. The Lifetime Impact Study for Achondroplasia (LISA), aimed to assess health-related quality of life and medical resource utilization among Latin America patients with achondroplasia.
Methods: Data were collected from individuals aged 3 years and above in Argentina, Brazil, and Colombia between 2018 and 2021.
Introduction: A recent study on patients with acute lower limb ischemia showed that the proportion of inadequate examination of lower extremity circulation was associated with higher rate of amputation and death. The aim of this systematic review was to explore evidence for how practical competence in performing a peripheral vascular status of the lower limb among medical students and junior doctors should be taught and examined.
Methods: The systematic review followed PRISMA guidelines and was published in PROSPERO.
Article Synopsis
- * Researchers administered a drug called PR-364 to mice after a heart attack, finding it significantly reduced mortality, preserved heart function, and slowed heart failure progression.
- * The findings revealed that PR-364 boosted the removal of damaged mitochondria and improved energy production in heart cells, suggesting it could be a promising treatment to protect heart tissue after a heart attack.
Objectives: This study evaluated the psychometric properties of the ceroid lipofuscinosis type 2 Quality of Life (CLN2 QoL) questionnaire.
Methods: Data from children with CLN2 disease aged 3-16 years receiving cerliponase alfa in the BMN 190-201 and BMN 190-202 clinical studies, collected via purposive sampling, were used to assess convergent and divergent validity, internal consistency and reliability. The clinically important difference (CID) was estimated with distribution- and anchor-based methods.