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
