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2024 2
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Filters applied: Meta-Analysis, Randomized Controlled Trial. Clear all
Page 1
AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trial.
Mendell JR, Muntoni F, McDonald CM, Mercuri EM, Ciafaloni E, Komaki H, Leon-Astudillo C, Nascimento A, Proud C, Schara-Schmidt U, Veerapandiyan A, Zaidman CM, Guridi M, Murphy AP, Reid C, Wandel C, Asher DR, Darton E, Mason S, Potter RA, Singh T, Zhang W, Fontoura P, Elkins JS, Rodino-Klapac LR. Mendell JR, et al. Nat Med. 2025 Jan;31(1):332-341. doi: 10.1038/s41591-024-03304-z. Epub 2024 Oct 9. Nat Med. 2025. PMID: 39385046 Free PMC article. Clinical Trial.
Delandistrogene moxeparvovec did not lead to a significant improvement in NSAA score at week 52. ...Safety was manageable and consistent with previous delandistrogene moxeparvovec trials. ClinicalTrials.gov: NCT05096221....
Delandistrogene moxeparvovec did not lead to a significant improvement in NSAA score at week 52. ...Safety was manageable and …
Quantitative Muscle Magnetic Resonance Outcomes in Patients With Duchenne Muscular Dystrophy: An Exploratory Analysis From the EMBARK Randomized Clinical Trial.
Vandenborne K, Walter GA, Straub V, Willcocks RJ, Forbes SC, Mercuri EM, Muntoni F, Ding K, Ennamuri S, Reid C, Murphy AP, Manfrini M, Mendell JR, Elkins JS, Rodino-Klapac LR. Vandenborne K, et al. JAMA Neurol. 2025 Jul 1;82(7):734-744. doi: 10.1001/jamaneurol.2025.0992. JAMA Neurol. 2025. PMID: 40354061 Free PMC article. Clinical Trial.
OBJECTIVE: To determine the effect of delandistrogene moxeparvovec on muscle quantitative magnetic resonance (QMR) measures of disease progression in patients in the EMBARK trial. ...Consistent with treatment effects on functional outcomes observed in the EMBARK tri …
OBJECTIVE: To determine the effect of delandistrogene moxeparvovec on muscle quantitative magnetic resonance (QMR) measures of …
Validity of remote live stream video evaluation of the North Star Ambulatory Assessment in patients with Duchenne muscular dystrophy.
P Lowes L, Alfano LN, Iammarino MA, Reash NF, Giblin K, Hu L, Yu L, Wang S, Salazar R, Mendell JR. P Lowes L, et al. PLoS One. 2024 May 16;19(5):e0300700. doi: 10.1371/journal.pone.0300700. eCollection 2024. PLoS One. 2024. PMID: 38753764 Free PMC article. Clinical Trial.
Participants in this analysis received delandistrogene moxeparvovec (as part of SRP-9001-101 [Study 101; NCT03375164] or SRP-9001-102 [Study 102; NCT03769116]) or were randomized to receive placebo (in Part 1 of Study 102). ...
Participants in this analysis received delandistrogene moxeparvovec (as part of SRP-9001-101 [Study 101; NCT03375164] or SRP-9 …