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Muscle Fat Replacement and Functional Decline in Neuromuscular Disorders – Quantitative Skeletal Muscle MRI in Clinical Trials

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    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).

    In this webinar, three experts will discuss various aspects of MRI biomarker development in NMD with a special focus on recent developments and experiences with MRI skeletal muscle biomarkers in DMD and FSHD:

    Sarah Sherlock, PhD, Pfizer – Experience of using MRI biomarkers in Duchenne Muscular Dystrophy trials

    Diego Cadavid, MD, Fulcrum Therapeutics – Opportunities and challenges with MRI biomarker development for FSHD drug trials

    Per Widholm, MD, AMRA Medical Research – Standardized whole-body MRI, precise monitoring of muscular dystrophies with heterogeneous pattern of onset and disease progression

    Webinar Date
    • 7 October 2020
    Sarah Sherlock, PhD
    Director Clinical and Translational Imaging
    Pfizer
    Read more
    Sarah Sherlock, PhD

    Director Clinical and Translational Imaging

    Pfizer

    Diego Cadavid, MD
    Senior Vice President, Clinical Development
    Fulcrum Therapeutics
    Read more
    Diego Cadavid, MD

    Senior Vice President, Clinical Development

    Fulcrum Therapeutics

    Per Widholm, MD
    Lead Scientist, MSK
    AMRA Medical Research
    Read more
    Per Widholm, MD

    Lead Scientist, MSK

    AMRA Medical Research

    What you will learn
    •
    How water and fat separated MRI has been used to describe disease stage and progression in neuromuscular disorders
    •
    Current examples of how quantitative MRI is used in neuromuscular trials
    •
    How whole-body MRI could improve the detection of treatment effect in NMD trials
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