P34 Reducing the cost of MRI in neuromuscular clinical trials: acceleration of fat-fraction measurement in Becker muscular dystrophy by combined compressed sensing and parallel imaging

2014 ◽  
Vol 24 ◽  
pp. S16
Author(s):  
K.G. Hollingsworth ◽  
D.M. Higgins ◽  
M. McCallum ◽  
L. Ward ◽  
A. Coombs ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Muscular Dystrophy ◽  
Compressed Sensing ◽  
Parallel Imaging ◽  
Becker Muscular Dystrophy ◽  
Fat Fraction ◽  
The Cost ◽  
Fraction Measurement

P.307Heterogeneous distribution of fatty infiltration - an important consideration in Becker muscular dystrophy clinical trials

2019 ◽  
Vol 29 ◽  
pp. S156-S157
Author(s):  
N. van de Velde ◽  
M. Hooijmans ◽  
Z. Koeks ◽  
J. van den Bergen ◽  
A. Sardjoe-Mishre ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Muscular Dystrophy ◽  
Fatty Infiltration ◽  
Becker Muscular Dystrophy

scholarly journals Dystrophin quantification and clinical correlations in Becker muscular dystrophy: implications for clinical trials

Brain ◽  
2011 ◽  
Vol 134 (12) ◽  
pp. 3547-3559 ◽  
Author(s):  
Karen Anthony ◽  
Sebahattin Cirak ◽  
Silvia Torelli ◽  
Giorgio Tasca ◽  
Lucy Feng ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Muscular Dystrophy ◽  
Becker Muscular Dystrophy ◽  
Clinical Correlations

scholarly journals Improving Highly Accelerated Fat Fraction Measurements for Clinical Trials in Muscular Dystrophy: Origin and Quantitative Effect of R2* Changes

Radiology ◽  
2015 ◽  
Vol 275 (2) ◽  
pp. 570-578 ◽  
Author(s):  
Thomas Loughran ◽  
David M. Higgins ◽  
Michelle McCallum ◽  
Anna Coombs ◽  
Volker Straub ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Muscular Dystrophy ◽  
Quantitative Effect ◽  
Fat Fraction

scholarly journals Challenges of Interpreting Dystrophin Content by Western Blot

US Neurology ◽  
2019 ◽  
Vol 15 (1) ◽  
pp. 40 ◽  
Author(s):  
Frederick J Schnell ◽  
Diane Frank ◽  
Sue Fletcher ◽  
Russell D Johnsen ◽  
Steve D Wilton ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Data Analysis ◽  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Western Blot ◽  
Becker Muscular Dystrophy ◽  
Human Muscle ◽  
Healthy Human ◽  
Dystrophin Protein ◽  
Western Blot Data

The Duchenne muscular dystrophy community has recently seen the first approved therapy for the restoration of dystrophin, based on its ability to increase levels of dystrophin protein, as determined by western blot. The approval, along with the initiation of clinical trials evaluating other dystrophin-restoring therapies, highlights the importance of accurate dystrophin quantitation. Nonoptimized western blot methods can reflect inaccurate results, especially in the quantitation of low dystrophin levels. A few key changes to standards and data analysis parameters can result in a low level of dystrophin (<0.5% of a healthy biopsy) being inaccurately interpreted as >20% of the levels reported in healthy human muscle. A review of the dystrophin western blot data on Duchenne and Becker muscular dystrophy biopsies is conducted, along with a thorough investigation of methodologies to quantify dystrophin.

scholarly journals Functional changes in Becker muscular dystrophy: implications for clinical trials in dystrophinopathies

2016 ◽  
Vol 6 (1) ◽  
Author(s):  
Luca Bello ◽  
Paola Campadello ◽  
Andrea Barp ◽  
Marina Fanin ◽  
Claudio Semplicini ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Muscular Dystrophy ◽  
Becker Muscular Dystrophy ◽  
Functional Changes

scholarly journals Baseline fat fraction is a strong predictor of disease progression in Becker muscular dystrophy

2022 ◽  
Author(s):  
Thom T. J. Veeger ◽  
Nienke M. Velde ◽  
Kevin R. Keene ◽  
Erik H. Niks ◽  
Melissa T. Hooijmans ◽  
...  
Keyword(s):  
Muscular Dystrophy ◽  
Disease Progression ◽  
Strong Predictor ◽  
Becker Muscular Dystrophy ◽  
Fat Fraction
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