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Home Neuroscience Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy

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Mar 16 / Roche and Genentech
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.
Neuroscience Duchenne Muscular Dystrophy MDA-2025

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Mar 16 / Roche and Genentech
3-Year Functional Outcomes of Patients With Duchenne Muscular Dystrophy: Pooled Delandistrogene Moxeparvovec Clinical Trial Data vs External Controls
We report 3-year functional outcomes of ambulatory patients (aged 4.0–8.9 years) with Duchenne muscular dystrophy treated with delandistrogene moxeparvovec, compared to propensity-score-matched external controls (EC). Data from 50 treated patients across multiple studies were analyzed using a propensity-score-weighted median regression model and a mixed-effects model for repeated measures. Results showed clinically meaningful stabilization of disease over 3 years in the treatment group versus the ECs, with smaller decline in the North Star Ambulatory Assessment scores and improved performance in timed function tests. These findings support the long-term stabilization or slowing of disease progression with delandistrogene moxeparvovec compared to ECs.
Neuroscience Duchenne Muscular Dystrophy MDA-2025

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Mar 16 / Roche and Genentech
Assessment of Cardiac Outcomes in Delandistrogene Moxeparvovec Clinical Trials for Duchenne Muscular Dystrophy
We report pooled cardiac safety outcomes from delandistrogene moxeparvovec studies with 1–5 years of follow-up in patients with Duchenne muscular dystrophy (DMD). Among 156 patients, two cases of myocarditis were resolved within three weeks, and on case of immune-mediated myositis stabilized within two weeks of treatment. Troponin I fluctuations were compatible with fluctuations observed in DMD. Long-term echocardiographic data showed stable left ventricular ejection fraction (LVEF), with no persistent cardiac injury. These findings indicate a manageable cardiac safety profile for delandistrogene moxeparvovec in this population of patients with DMD.
Neuroscience Duchenne Muscular Dystrophy MDA-2025

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Mar 16 / Roche and Genentech
Muscle MRI outcomes in patients with Duchenne Muscular Dystrophy treated with delandistrogene moxeparvovec: Findings from EMBARK Part 1
We report up to 2-year exploratory analyses of muscle health outcomes and changes in muscle pathology assessed by MR (magnetic resonance) in EMBARK Part 1 to evaluate the effect of delandistrogene moxeparvovec on DMD disease progression. At Week 52, MR measures improved in the delandistrogene moxeparvovec group compared with the placebo group, suggesting stabilization or less progression of muscle pathology with treatment, with statistical significance across the 12 MR parameters. At Week 104, MRI changes generally favored delandistrogene moxeparvovec vs. Week 52 placebo, even though some degree of progression of muscle pathology was observed. Ongoing analyses will assist with quantifying signs of continued long-term benefits for muscle pathology.
Neuroscience Duchenne Muscular Dystrophy MDA-2025

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Mar 16 / Roche and Genentech
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.
Neuroscience Duchenne Muscular Dystrophy MDA-2025

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Mar 16 / Roche and Genentech
Longitudinal Stride-Level Evaluation of Ambulatory Function with Ankle Wearable Technology in Ambulant DMD Patients Below 4 Years Old
We report on a multicenter study assessing the feasibility, robustness, and sensitivity of using stride velocity 95th percentile (SV95C) to evaluate ambulatory function in patients with DMD under four years of age. Syde provided reliable, stride-level data, with SV95C distinguishing patients with DMD from age-matched controls. Early findings suggest that SV95C improves after steroid initiation and may capture early disease progression. Good adherence and high reliability support the use of digital biomarkers in this young population.
Neuroscience Duchenne Muscular Dystrophy MDA-2025
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  • Duchenne Muscular Dystrophy
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Nov 5 / Roche
Roche Educational Webinar: DMD highlights from the 2024 WMS Annual Congress
Non-promotional webinar intended to: - Provide an overview of the clinical and scientific Duchenne muscular dystrophy (DMD) highlights from the 29th International WMS Annual Congress - Explore the recent advances in assessing progression and outcomes in patients living with DMD, including MRI and wearable technologies - Discuss recent findings from DMD clinical trials with a focus on AAV-based gene therapy.
Neuroscience Duchenne Muscular Dystrophy
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March 16 - March 19, 2025 / Dallas, TX
MDA 2025
Neuroscience Duchenne Muscular Dystrophy Spinal Muscular Atrophy
Subject:
Muscular Dystrophy Association - 7th Clinical and Scientific Conference 2025
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This website is intended for healthcare professionals outside the United Kingdom (UK) and Australia. Registration status and prescribing information of medicinal products may differ between countries. Please refer to local product information for any medicinal products mentioned on this website.