Expert Interview
Looking at Sarepta's confirmatory trial of Vyondys 53 and Amonydys 45 exon skippers for Duchenne muscular dystrophy
Ticker(s): SRPTInstitution: University of Oxford
Professor of Paediatric Neuromuscular Diseases at the MDUK Oxford Neuromuscular Centre and Invited Professor of Child Neurology at Liège University, specializing in Duchenne muscular dystrophy (DMD) and spinal muscular atrophy (SMA).
Leads international clinical trial networks and mentors paediatric neurology fellows, serving as principal investigator for multiple gene therapy and disease-modifying trials in DMD and SMA.
Pioneers research on innovative outcome measures, including wearable sensors and real-life digital biomarkers, and leads the newborn screening program for SMA in southern Belgium, with over 150 publications advancing neuromuscular clinical research.
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