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Myology 2022

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Apr 13 / Roche
Myology 2022 Symposium: Heading to the future of clinical research in neuromuscular disorders
Watch the Myology 2022 symposium, where our Chair, Professor Francesco Muntoni (UK) alongside Charlotte Lilien (UK) and Guillaume Millet (France), explore the current treatment landscape, unmet needs and emerging therapies in SMA and DMD, and discuss appropriate treatment outcomes and objective physiological biomarkers in heterogeneous NMD patient populations.

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Sep 12 / Roche and Genentech
Integrated Analyses of Data from Clinical Trials of Delandistrogene Moxeparvovec in DMD
Delandistrogene moxeparvovec (SRP-9001) is an investigational gene transfer therapy developed for targeted skeletal and cardiac muscle expression of micro-dystrophin (a shortened, functional dystrophin protein) that is being studied in patients with Duchenne muscular dystrophy (DMD). The objective of this integrated analysis is to evaluate functional data from patients (≥4 to ≤8 years old) with DMD who have participated in the delandistrogene moxeparvovec studies and compare these data with a control cohort of propensity-matched external control patients. Here we present an integrated analysis of functional data from the 52 patients assessed to date from Study 101 (N=4), Parts 1 and 2 of Study 102 (n=28) who received the target dose (1.33x1014 vg/kg by linear qPCR) of delandistrogene moxeparvovec over 1 year, and patients from Cohort 1 of ENDEAVOR (n=20). Collective safety data from all patients in Study 101, Study 102, and all cohorts of ENDEAVOR (N=84) are also presented.

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Sep 12 / Roche and Genentech
One-year ENDEAVOR Data (Ambulatory, ≥4 to <8-year-olds): Phase 1b Trial of Delandistrogene Moxeparvovec in DMD
Delandistrogene moxeparvovec (SRP-9001) is an investigational gene transfer therapy developed for targeted skeletal and cardiac muscle expression of micro-dystrophin (a shortened, functional dystrophin protein) that is being studied in patients with Duchenne muscular dystrophy (DMD). ENDEAVOR (NCT04626674) is a two-part, open-label, Phase 1b study assessing the expression and safety (over 260 weeks) of commercially representative delandistrogene moxeparvovec material in four cohorts of boys with DMD. We present 1-year safety and functional data and 12-week expression data from all patients in Cohort 1 (n=20) following treatment with commercially representative delandistrogene moxeparvovec material.