The recent development of quantitative skeletal MRI biomarkers, describing the extent of muscle atrophy and muscle fat infiltration and replacement, has enabled the detection and measurement of NMD earlier and more precisely compared to clinical endpoints.
TORONTO (PRWEB) September 25, 2020
Drug development in neuromuscular disorders needs robust biomarker and clinical endpoints to study drug efficacy in early and late stage trials. Ultimately, functional decline and motor disability are the main clinical manifestations of neuromuscular disorders (NMD) and used for approval of novel therapeutics. However, assessment of changes in neurological function is associated with large variability and many NMDs are slowly progressive, thus limiting its feasibility as efficacy endpoint in early clinical development.
One of the most promising efficacy biomarkers in neuromuscular disorders for early clinical development is skeletal muscle MRI. The recent development of quantitative skeletal MRI biomarkers, describing the extent of muscle atrophy and muscle fat infiltration and replacement, has enabled the detection and measurement of NMD earlier and more precisely compared to clinical endpoints. Standardized quantitative skeletal muscle MRI has been used to objectively evaluate NMD across different disease stages and measure longitudinal changes in diseases with complex onset patterns and slow progression such as FSHD (facioscapulomuscular dystrophy) and DMD (Duchenne Muscular Dystrophy).
Join Sarah Sherlock, PhD, Director Clinical and Translational Imaging, Pfizer, Diego Cadavid, MD, Senior Vice President, Clinical Development, Fulcrum Therapeutics and Per Widholm, MD, Lead Scientist, MSK, AMRA Medical Research in a live webinar on Wednesday, October 7, 2020 at 11am EDT (4pm BST/UK).
For more information or to register for this event, visit Muscle Fat Replacement and Functional Decline in Neuromuscular Disorders – Quantitative Skeletal Muscle MRI in Clinical Trials.
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