A new study published in JAMA Neurology revealed promising evidence that the gene therapy delandistrogene moxeparvovec (Elevidys) may help slow muscle degeneration in boys with Duchenne muscular dystrophy (DMD).
The analysis included 39 boys aged 4 to 8 with DMD, half of whom received the gene therapy and the other half a placebo. The researchers found that boys treated with delandistrogene moxeparvovec showed significantly less fat replacement and tissue damage in key leg muscles compared to those given the placebo. The findings were based on quantitative magnetic resonance (QMR), an advanced imaging technique, which measured changes in muscle fat fraction and tissue health over a one-year period. Previous research has linked fat fraction to loss of ambulation (the ability to walk) in children with DMD.
Delandistrogene moxeparvovec aims to slow the progression of the rare muscular disorder by using a modified virus to deliver a shortened version of the dystrophin gene — the protein that is mutated or missing in individuals with DMD.
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The new data comes from the subset of boys in the EMBARK Phase 3 clinical trial who underwent muscle QMR imaging. The EMBARK trial included 125 participants total and did not meet its primary goal of a meaningful motor function improvement. However, key secondary measures such as time to rise and walking speed did show some improvement with the gene therapy.
The new imaging results from a subset of patients from this study add to the evidence supporting the use of delandistrogene moxeparvovec to treat DMD, the researchers say.
“Consistent with treatment effects on functional outcomes observed in the EMBARK trial, these results suggest stabilization or less progression of muscle pathology with delandistrogene moxeparvovec — adding to the totality of evidence supporting disease stabilization or slowing of disease progression with delandistrogene moxeparvovec,” they concluded.
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