Delandistrogene moxeparvovec (also known as ELEVIDYS) is a recombinant gene therapy used for the treatment of Duchenne muscular dystrophy, developed by Sarepta Therapeutics and acquired by Roche.
Delandistrogene moxeparvovec
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Approval process
It is designed to deliver into the body a gene that leads to production of micro-dystrophin that contains selected domains of the dystrophin protein present in normal muscle cells.
In January 2024 the National Institute of Health and Care Excellence (NICE) opened the scoping stage of an appraisal for the treatment. This is the very start of the NICE appraisal process, and once completed NICE will publish further information about the full appraisal process and timeline.
Support and information
Duchenne muscular dystrophy
Duchenne muscular dystrophy is a muscle wasting condition caused by the lack of a protein called dystrophin. It usually affects only boys.
Research
We fund groundbreaking research to learn more about muscle wasting conditions and lead us to new treatments. We’ve already made advances that would have been unthinkable just 10 years ago, and we are determined to go even further and faster.
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