ALSFRS-R is a tool designed to measure disease progress in ALS patients. It consists of 12 items grouped into four functions designed to assess disabilities according to the Activities of daily living (ADL). Our objective was to validate the Spanish version of ALSFRS-R based on the original version. Four examiners assessed 73 ALS patients, applying the ALSFRS-R, ALSAQ-40 and the respiratory function variable assessed by the SRI scale, which measures respiratory insufficiency. Internal consistency and test-retest correlations were measured using Cronbach's alpha and Spearman's Rho tests. Factor analysis was performed by applying Varimax rotation and Kaiser standardization. Validity was analysed based on correlations between items in the ALSFRS-R scales and equivalents in the ALSAQ-40 and SRI questionnaires. The results showed high internal consistency (0.77-0.95) and a good test-retest correlation (0.80-0.95). Factor analysis showed a 73.3% principal component contribution; the weight of each item regarding their corresponding factors was 0.7-0.9. High correlations were observed (rs >0.60) between corresponding factors of ALSFRS-R/ALSAQ-40 and ALSFRS-R/SRI. We conclude that the version obtained from the ALSFRS-R maintains the internal consistency and validity of the construct of the original scale. The Spanish version of ALSFRS-R is available for readers at http://www.fundela.es/verOtras.php.
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http://dx.doi.org/10.3109/17482968.2010.489115 | DOI Listing |
Clin Drug Investig
December 2024
Queensland Brain Institute, University of Queensland, St Lucia, Brisbane, QLD, 4072, Australia.
Background: Erythropoietin-producing hepatocellular carcinoma A4 (EphA4) is implicated in the pathophysiology of amyotrophic lateral sclerosis. EphA4 fusion protein (EphA4-Fc) inhibits EphA4 function in vivo but is too short-lived for prolonged therapy. NUN-004 (mEphA4-Fc) is a modified EphA4-Fc engineered for an extended half-life.
View Article and Find Full Text PDFOrphanet J Rare Dis
July 2024
Department of Neurology, Peking University Third Hospital, 49 North Garden Road, Haidian District, Beijing, 100191, China.
Objective: The Center for Neurologic Study Bulbar Function Scale (CNS-BFS) was specifically designed as a self-reported measure of bulbar function. The purpose of this research was to validate the Chinese translation of the CNS-BFS as an effective measurement for the Chinese population with ALS.
Methods: A total of 111 ALS patients were included in this study.
Medicine (Baltimore)
June 2024
Department of Neurology, School of Medicine, Kyungpook National University, Kyungpook National University Chilgok Hospital, Daegu, South Korea.
Neurology
July 2024
From the UMC Utrecht Brain Center (R.P.A.v.E., L.H.v.d.B.), University Medical Center Utrecht, the Netherlands; and Department of Biomedical Data Science (Y.L.), Stanford University, CA.
Background And Objectives: The Patient-Ranked Order of Function (PROOF) is a novel approach to account for patient-reported preferences in the evaluation of treatments of amyotrophic lateral sclerosis (ALS). In this study, we assess the reliability and prognostic value of different sets of patient-reported preferences that can be used for the PROOF end point.
Methods: Data were obtained through online surveys over the course of 12 months using the population-based registry of the Netherlands.
Value Health
October 2024
US Food and Drug Administration, Silver Spring, Maryland, USA.
Objectives: In 2021, the US Congress passed the Accelerating Access to Critical Therapies for Amyotrophic Lateral Sclerosis Act. The law encourages development of "tools, methods, and processes" to improve clinical trial efficiency for neurodegenerative diseases. The Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) is an outcome measure administered during in-person clinic visits and used to support investigational studies for persons living with amyotrophic lateral sclerosis.
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