scholarly journals A New Functional Scale and Ambulatory Functional Classification of Duchenne Muscular Dystrophy: Scale Development and Preliminary Analyses of Reliability and Validity

2018 ◽  
Vol 42 (5) ◽  
pp. 690-701
Author(s):  
Jungyoon Kim ◽  
Il-Young Jung ◽  
Sang Jun Kim ◽  
Joong-Yub Lee ◽  
Sue Kyung Park ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Scale Development ◽  
Reliability And Validity ◽  
Functional Classification ◽  
Functional Scale

Performance of the Upper Limb Module—A Reliable and Valid Evaluation for Chinese Patients with Duchenne Muscular Dystrophy

2020 ◽  
Author(s):  
Yuen Yee Alice Chiu ◽  
Chun Wai Lo ◽  
Chi Kuk Connie Hui ◽  
Wai Chong Susanna Choi ◽  
So Lun Lee ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Upper Limb ◽  
Forced Vital Capacity ◽  
Motor Performance ◽  
Vital Capacity ◽  
High Reliability ◽  
Reliability And Validity ◽  
Chinese Patients ◽  
Rater Reliability

Abstract Background Duchenne muscular dystrophy is a genetic disease leading to progressive muscle weakness and degeneration. Effective assessment tool is needed to allow monitoring of progress to guide the management. This study assessed the reliability and validity of the Performance of Upper Limb (PUL) Module when used in patients with Duchenne Muscular Dystrophy (DMD). MethodsTotal thirty-three Chinese DMD patients were included. Twenty-five video-recorded PUL Module version 1.3 assessments were performed for the recruited patients with three raters evaluated the same recorded video for inter-rater reliability and evaluated the same performance one month later for intra-rater reliability. Construct validity was assessed correlating the PUL Module scores with the patients’ age, their forced vital capacity (N=25) and their Hammersmith motor scale scores (N=25) performed on the same day. ResultsThe intra-rater and inter-rater reliability (ICC 0.92 - 0.99), internal consistency (Cronbach’s alpha 0.97 - 0.99) and known groups validity (AUC 0.97) of PUL module were excellent. PUL was negatively correlated with age (r = -0.912), and positively correlated with the forced vital capacity (r = 0.87) and the Hammersmith motor scale (r = 0.84). The findings confirm the high reliability and validity of PUL module, and its high clinical relevancy in monitoring the deteriorating upper limb motor performance that strongly correlated with the lung function and generalized motor performance as age increased in DMD. ConclusionThis first study of PUL module in Chinese patients with DMD confirmed that it is a reliable valid tool to monitor clinical progress and outcome for DMD.

scholarly journals Cognitive Deficits in Myopathies

2020 ◽  
Vol 21 (11) ◽  
pp. 3795
Author(s):  
Eleni Peristeri ◽  
Athina-Maria Aloizou ◽  
Paraskevi Keramida ◽  
Zisis Tsouris ◽  
Vasileios Siokas ◽  
...  
Keyword(s):  
Skeletal Muscle ◽  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Brain Imaging ◽  
Cognitive Deficits ◽  
Wide Spectrum ◽  
Comprehensive Overview ◽  
Clinical Observations ◽  
Abnormal Structure

Myopathies represent a wide spectrum of heterogeneous diseases mainly characterized by the abnormal structure or functioning of skeletal muscle. The current paper provides a comprehensive overview of cognitive deficits observed in various myopathies by consulting the main libraries (Pubmed, Scopus and Google Scholar). This review focuses on the causal classification of myopathies and concomitant cognitive deficits. In most studies, cognitive deficits have been found after clinical observations while lesions were also present in brain imaging. Most studies refer to hereditary myopathies, mainly Duchenne muscular dystrophy (DMD), and myotonic dystrophies (MDs); therefore, most of the overview will focus on these subtypes of myopathies. Most recent bibliographical sources have been preferred.

Evaluation Scale Development, Reliability for Sitting and Standing From the Chair for Duchenne Muscular Dystrophy

2013 ◽  
Vol 45 (2) ◽  
pp. 117-126 ◽  
Author(s):  
Michele Emy Hukuda ◽  
Renata Escorcio ◽  
Lilian Aparecida Yoshimura Fernandes ◽  
Eduardo Vital de Carvalho ◽  
Fátima Aparecida Caromano
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Scale Development

scholarly journals Classification of the Gait Patterns of Boys With Duchenne Muscular Dystrophy and Their Relationship to Function

2010 ◽  
Vol 25 (9) ◽  
pp. 1103-1109 ◽  
Author(s):  
Susan Sienko Thomas ◽  
Cathleen E. Buckon ◽  
Alina Nicorici ◽  
Anita Bagley ◽  
Craig M. McDonald ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Gait Patterns

scholarly journals Vamorolone trial in Duchenne muscular dystrophy shows dose-related improvement of muscle function

Neurology ◽  
2019 ◽  
Vol 93 (13) ◽  
pp. e1312-e1323 ◽  
Author(s):  
Eric P. Hoffman ◽  
Benjamin D. Schwartz ◽  
Laurel J. Mengle-Gaw ◽  
Edward C. Smith ◽  
Diana Castro ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Natural History ◽  
Muscle Function ◽  
Open Label ◽  
Suspension Formulation ◽  
Efficacy Outcome ◽  
Dose Study ◽  
Dose Levels

ObjectiveTo study vamorolone, a first-in-class steroidal anti-inflammatory drug, in Duchenne muscular dystrophy (DMD).MethodsAn open-label, multiple-ascending-dose study of vamorolone was conducted in 48 boys with DMD (age 4–<7 years, steroid-naive). Dose levels were 0.25, 0.75, 2.0, and 6.0 mg/kg/d in an oral suspension formulation (12 boys per dose level; one-third to 10 times the glucocorticoid dose in DMD). The primary goal was to define optimal doses of vamorolone. The primary outcome for clinical efficacy was time to stand from supine velocity.ResultsOral administration of vamorolone at all doses tested was safe and well tolerated over the 24-week treatment period. The 2.0–mg/kg/d dose group met the primary efficacy outcome of improved muscle function (time to stand; 24 weeks of vamorolone treatment vs natural history controls), without evidence of most adverse effects of glucocorticoids. A biomarker of bone formation, osteocalcin, increased in vamorolone-treated boys, suggesting possible loss of bone morbidities seen with glucocorticoids. Biomarker outcomes for adrenal suppression and insulin resistance were also lower in vamorolone-treated patients with DMD relative to published studies of glucocorticoid therapy.ConclusionsDaily vamorolone treatment suggested efficacy at doses of 2.0 and 6.0 mg/kg/d in an exploratory 24-week open-label study.Classification of evidenceThis study provides Class IV evidence that for boys with DMD, vamorolone demonstrated possible efficacy compared to a natural history cohort of glucocorticoid-naive patients and appeared to be tolerated.

The reliability and validity of Turkish version of pedsQL multidimensional fatigue scale in Duchenne muscular dystrophy

2017 ◽  
Vol 27 ◽  
pp. S235
Author(s):  
I. Alemdaroğlu ◽  
N. Bulut ◽  
S. Bozgeyik ◽  
A. Karaduman ◽  
H. Topaloğlu ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Reliability And Validity ◽  
Turkish Version ◽  
Multidimensional Fatigue Scale
Sign in / Sign up

Export Citation Format

Share Document