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

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

Article  CAS  PubMed  Google Scholar 

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

Article  Google Scholar 

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

Article  CAS  PubMed  PubMed Central  Google Scholar 

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

Article  CAS  PubMed  PubMed Central  Google Scholar 

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

Article  CAS  PubMed  PubMed Central  Google Scholar 

Birnkrant DJ, Bushby K, Bann CM et al (2018a) Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and Gastrointestinal and nutritional management. Lancet Neurol 17:251–267. https://doi.org/10.1016/S1474-4422(18)30024-3

Article  PubMed  PubMed Central  Google Scholar 

Birnkrant DJ, Bushby K, Bann CM et al (2018b) Diagnosis and management of Duchenne muscular dystrophy, part 2: respiratory, cardiac, bone health, and orthopaedic management. Lancet Neurol 17:347–361. https://doi.org/10.1016/S1474-4422(18)30025-5

Article  PubMed  PubMed Central  Google Scholar 

Bladen CL, Salgado D, Monges S et al (2015) The TREAT-NMD DMD global database: analysis of more than 7,000 Duchenne muscular dystrophy mutations. Hum Mutat 36:395–402. https://doi.org/10.1002/humu.22758

Article  CAS  PubMed  Google Scholar 

Davies KE, Guiraud S (2019) Micro-dystrophin genes bring hope of an effective therapy for Duchenne muscular dystrophy. Mol Ther 27:486–488. https://doi.org/10.1016/j.ymthe.2019.01.019

Article  CAS  PubMed  PubMed Central  Google Scholar 

DerSimonian R, Laird N (1986) Meta-analysis in clinical trials. Control Clin Trials 7:177–188. https://doi.org/10.1016/0197-2456(86)90046-2

Article  CAS  PubMed  Google Scholar 

Duvuru R, Neumann S, Beemarajan E et al (2025) Acute liver injury following delandistrogene Moxeparvovec gene therapy requiring intravenous Immunoglobulin. Pediatr Neurol 163:1–3. https://doi.org/10.1016/j.pediatrneurol.2024.11.005

Article  PubMed  Google Scholar 

Egger M, Smith GD, Schneider M, Minder C (1997) Bias in meta-analysis detected by a simple, graphical test. BMJ 315:629–634. https://doi.org/10.1136/bmj.315.7109.629

Article  CAS  PubMed  PubMed Central  Google Scholar 

Elhalag RH, Motawea KR, Talat NE et al (2023) Efficacy of Vamorolone in treatment of Duchene muscle dystrophy. A meta-analysis. Front Neurol 14:1107474

Article  PubMed  PubMed Central  Google Scholar 

Emery AE (1991) Population frequencies of inherited neuromuscular diseases–a world survey. Neuromuscul Disord 1:19–29. https://doi.org/10.1016/0960-8966(91)90039-u

Article  CAS  PubMed  Google Scholar 

Ertl HCJ (2022) Immunogenicity and toxicity of AAV gene therapy. Front Immunol 13:975803. https://doi.org/10.3389/fimmu.2022.975803

Article  CAS  PubMed  PubMed Central  Google Scholar 

Food and Drug Administration (2024) Highlights of prescribing information. https://www.fda.gov/media/169679/download. Accessed 2 Mar 2025

Gambetta KE, McCulloch MA, Lal AK et al (2022) Diversity of dystrophin gene mutations and disease progression in a contemporary cohort of Duchenne muscular dystrophy. Pediatr Cardiol 43:855–867. https://doi.org/10.1007/s00246-021-02797-6

Article  PubMed  Google Scholar 

Guyatt G, Oxman AD, Akl EA et al (2011) GRADE guidelines: 1. Introduction-GRADE evidence profiles and summary of findings tables. J Clin Epidemiol 64:383–394. https://doi.org/10.1016/j.jclinepi.2010.04.026

Article  PubMed  Google Scholar 

Harper AD, Topaloglu H, Mercuri E et al (2024) Safety and efficacy of Viltolarsen in ambulatory and nonambulatory males with Duchenne muscular dystrophy. Sci Rep 14:23488. https://doi.org/10.1038/s41598-024-70783-y

Article  CAS  PubMed  PubMed Central  Google Scholar 

Higgins JPT, Thompson SG (2002) Quantifying heterogeneity in a meta-analysis. Stat Med 21:1539–1558. https://doi.org/10.1002/sim.1186

Article  PubMed  Google Scholar 

Higgins JPT, Thomas J, Chandler J et al (2019) Cochrane handbook for systematic reviews of interventions. Cochrane Handbook for Systematic Reviews of Interventions 1–694. https://doi.org/10.1002/9781119536604

Horn S, Fehse B (2024) How safe is gene therapy? Second death after Duchenne therapy; [Wie sicher ist die Gentherapie? Zweiter Todesfall nach Therapie der Duchenne-Muskeldystrophie]. Innere Medizin (Germany) 65:617–623. https://doi.org/10.1007/s00108-024-01711-5

Article  Google Scholar 

Hoy SM (2023) Delandistrogene moxeparvovec: first approval. Drugs 83:1323–1329. https://doi.org/10.1007/s40265-023-01929-x

Article  CAS  PubMed  Google Scholar 

Jolly H, Aartsma-Rus A, Bertini E et al (2025) Gene therapy approval for Duchenne muscular dystrophy: a European perspective. Lancet 405:1572–1573

Article  PubMed  Google Scholar 

Kipke J, Birnkrant DJ, Jin JB et al (2021) A systematic review of Pharmacologic therapies for the cardiomyopathy of Duchenne muscular dystrophy. Pediatr Pulmonol 56:782–795. https://doi.org/10.1002/ppul.25261

Article  PubMed  Google Scholar 

Lim KRQ, Nguyen Q, Yokota T (2020) Genotype-phenotype correlations in Duchenne and Becker muscular dystrophy patients from the Canadian neuromuscular disease registry. J Pers Med 10:1–18. https://doi.org/10.3390/jpm10040241

Article  Google Scholar 

Ma L-L, Wang Y-Y, Yang Z-H et al (2020) Methodological quality (risk of bias) assessment tools for primary and secondary medical studies: what are they and which is better? Mil Med Res 7:7. https://doi.org/10.1186/s40779-020-00238-8

Article  PubMed  PubMed Central  Google Scholar 

Mah JK, Korngut L, Dykeman J et al (2014) A systematic review and meta-analysis on the epidemiology of Duchenne and Becker muscular dystrophy. Neuromuscul Disord 24:482–491. https://doi.org/10.1016/j.nmd.2014.03.008

Article  PubMed  Google Scholar 

Marden FA, Connolly AM, Siegel MJ, Rubin DA (2005) Compositional analysis of muscle in boys with Duchenne muscular dystrophy using MR imaging. Skeletal Radiol 34:140–148. https://doi.org/10.1007/s00256-004-0825-3

Article  PubMed  Google Scholar 

McDonald CM, Abresch RT, Carter GT et al (1995) Profiles of neuromuscular diseases. Duchenne muscular dystrophy. Am J Phys Med Rehabil 74:S70–92. https://doi.org/10.1097/00002060-199509001-00003

Article  CAS  PubMed  Google Scholar 

Mendell JR, Sahenk Z, Lehman K et al (2020) Assessment of systemic delivery of rAAVrh74.MHCK7.micro-dystrophin in children with Duchenne muscular dystrophy: A nonrandomized controlled trial. JAMA Neurol 77:1122–1131. https://doi.org/10.1001/jamaneurol.2020.1484

Article  PubMed 

Comments (0)

No login
gif