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BIOIMAGE-NMD will develop and apply imaging technology to monitor response to novel therapies in neuromuscular diseases (NMD) and will use Duchenne muscular dystrophy (DMD) as an exemplar disease. DMD is well characterised genetically and clinically but to date a disease modifying treatment is not available. One of the most promising developments for future treatment of NMD is RNA modulation throu ...

Duchenne muscular dystrophy (DMD) is a progressive, lethal muscle degenerative condition arising from the absence of dystrophin in skeletal and cardiac muscles. 65% of DMD boys have out-of-frame deletions. Modulation of pre-mRNA splicing by exon skipping is the most promising molecular intervention in DMD. 2 Phase Ib and 2 Phase IIa clinical trials (MDEX Consortium in collaboration with Sarepta Th ...