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Review
. 2025 Aug;144(8):795-807.
doi: 10.1007/s00439-025-02758-6. Epub 2025 Jun 26.

Efficacy of delandistrogene moxeparvovec on Duchenne muscular dystrophy: a systematic review and meta-analysis

Carlos Pascual-Morena  1   2 , Silvana Patiño-Cardona  1 , Irene Martínez-García  3 , Jaime Fernández-Bravo-Rodrigo  4 , Miriam Garrido-Miguel  1   2
Affiliations
Review

Efficacy of delandistrogene moxeparvovec on Duchenne muscular dystrophy: a systematic review and meta-analysis

Carlos Pascual-Morena et al. Hum Genet. 2025 Aug.
No abstract available

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Conflict of interest statement

Declarations. Competing interests: The authors declare no competing interests.

References

    1. Aartsma-Rus A, Ginjaar IB, Bushby K (2016) The importance of genetic diagnosis for Duchenne muscular dystrophy. J Med Genet 53:145–151. https://doi.org/10.1136/jmedgenet-2015-103387 - DOI - PubMed
    1. Alfano LN, Lowes LP, Berry KM et al (2014) T.P.1: pilot study evaluating motivation on the performance of timed walking in boys with Duchenne muscular dystrophy. Neuromuscul Disord 24:860. https://doi.org/10.1016/j.nmd.2014.06.224 - DOI
    1. Arora H, Willcocks RJ, Lott DJ et al (2018) Longitudinal timed function tests in Duchenne muscular dystrophy: imagingdmd cohort natural history. Muscle Nerve 58:631–638. https://doi.org/10.1002/mus.26161 - DOI - PubMed - PMC
    1. Asher D, Dai D, Klimchak AC et al (2023) Paving the way for future gene therapies: A case study of scientific spillover from delandistrogene Moxeparvovec. Mol Ther Methods Clin Dev 30:474–483. https://doi.org/10.1016/j.omtm.2023.08.002 - DOI - PubMed - PMC
    1. Ayyar Gupta V, Pitchforth JM, Domingos J et al (2023) Determining minimal clinically important differences in the North star ambulatory assessment (NSAA) for patients with Duchenne muscular dystrophy. PLoS ONE 18:e0283669. https://doi.org/10.1371/journal.pone.0283669 - DOI - PubMed - PMC

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