Duchenne Muscular Dystrophy

Muscle MRI outcomes in patients with Duchenne Muscular Dystrophy treated with delandistrogene moxeparvovec: Findings from EMBARK Part 1

We report exploratory analyses of muscle health outcomes and changes in muscle pathology assessed by MR (magnetic resonance) in a subset of patients in EMBARK to evaluate the effect of delandistrogene moxeparvovec on DMD disease progression. At Week 52, MR measures were improved with delandistrogene moxeparvovec versus placebo, suggesting stabilization or less progression of muscle pathology. At Week 104, MRI changes generally favored delandistrogene moxeparvovec versus Week 52 placebo. Additional analyses will continue to assess long-term benefits in muscle pathology.

Cardiac MRI Outcomes in Patients with Duchenne Muscular Dystrophy Treated with Delandistrogene Moxeparvovec: Findings from EMBARK Part 1

We report exploratory analyses of cardiac outcomes assessed by MRI (magnetic resonance imaging) in a subset of patients in EMBARK to evaluate the effect of delandistrogene moxeparvovec on cardiac function, volume, and mass. At Week 52 and 104, there were no signs of alterations in cardiac measures versus placebo. Results indicate no adverse cardiac effects 2 years after treatment. Longer-term cardiac safety and efficacy will continue to be assessed.

Long-Term Functional Outcomes and Safety of Delandistrogene Moxeparvovec in DMD: EMBARK 2-Year and Pooled 3-Year Analyses

We report long-term functional outcomes from delandistrogene moxeparvovec clinical studies: EMBARK 2-year functional and safety data and Week 64 biological data, and pooled 3‑year analyses from Study 101, Study 102 and ENDEAVOR Cohort 1. Results indicate stabilization or slowing of disease progression compared with well-matched external controls in functional outcomes prognostic for delaying loss of ambulation, and long-term safety was consistent with prior experience.

Muscle MRI outcomes in patients with Duchenne Muscular Dystrophy treated with delandistrogene moxeparvovec: Findings from EMBARK Part 1

We report 1-year exploratory analyses of muscle health outcomes and changes in muscle pathology assessed by MRI (magnetic resonance imaging) in EMBARK Part 1 to evaluate the effect of delandistrogene moxeparvovec on DMD disease progression. Overall results indicate that the MRS (magnetic resonance spectroscopy)- and MRI-measured fat fraction, and MRI T2 (transverse relaxation time) were reduced in the delandistrogene moxeparvovec versus the placebo group at Week 52. This suggests stabilization or slowing of disease progression with treatment, which aligns with the secondary functional outcomes observed in this study. MR results demonstrated nominally statistically significant differences between the treatment and placebo groups across muscle regions and MR parameters.

Long-Term Safety and Tolerability of Delandistrogene Moxeparvovec in Duchenne Muscular Dystrophy: Phase 1 to Phase 3 Clinical Trials

We report pooled safety outcomes from clinical trials of delandistrogene moxeparvovec in Duchenne muscular dystrophy (DMD) with up to 5 years of follow-up. Among 156 patients, common treatment-related treatment-emergent adverse events (≥15%) included vomiting, nausea, decreased appetite and increased glutamate dehydrogenase, and upper abdominal pain, mostly resolving spontaneously. Treatment-related serious adverse events included liver abnormalities, rhabdomyolysis, vomiting, myocarditis, immune-mediated myositis, nausea, and pyrexia. Safety was consistent across ambulatory and non-ambulatory patients, with no deaths, study discontinuations, or complement activation-related adverse events. These findings support the manageable safety and tolerability profile of delandistrogene moxeparvovec across a broad population of patients with DMD.

Long-term functional outcomes, safety, and micro-dystrophin expression following delandistrogene moxeparvovec treatment in DMD: EMBARK 2-year results

We report 2-year functional and safety outcomes and Week 64 biological outcomes from patients treated with delandistrogene moxeparvovec in Part 1 of EMBARK. Results indicate stabilization or slowing of disease progression compared with well-matched external controls in functional outcomes prognostic for delaying loss of ambulation, with nominally statistically significant differences between the treatment group and external control cohort. Sustained micro‑dystrophin expression and localization to the sarcolemma was observed up to Week 64, and safety was consistent with prior experience.

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Duchenne Muscular Dystrophy

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