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Page 1
Gene therapy with bidridistrogene xeboparvovec for limb-girdle muscular dystrophy type 2E/R4: phase 1/2 trial results.
Mendell JR, Pozsgai ER, Lewis S, Griffin DA, Lowes LP, Alfano LN, Lehman KJ, Church K, Reash NF, Iammarino MA, Sabo B, Potter R, Neuhaus S, Li X, Stevenson H, Rodino-Klapac LR. Mendell JR, et al. Among authors: potter r. Nat Med. 2024 Jan;30(1):199-206. doi: 10.1038/s41591-023-02730-9. Epub 2024 Jan 4. Nat Med. 2024. PMID: 38177855 Free PMC article. Clinical Trial.
Long-term safety and functional outcomes of delandistrogene moxeparvovec gene therapy in patients with Duchenne muscular dystrophy: A phase 1/2a nonrandomized trial.
Mendell JR, Sahenk Z, Lehman KJ, Lowes LP, Reash NF, Iammarino MA, Alfano LN, Lewis S, Church K, Shell R, Potter RA, Griffin DA, Hogan M, Wang S, Mason S, Darton E, Rodino-Klapac LR. Mendell JR, et al. Among authors: potter ra. Muscle Nerve. 2024 Jan;69(1):93-98. doi: 10.1002/mus.27955. Epub 2023 Aug 14. Muscle Nerve. 2024. PMID: 37577753 Clinical Trial.
Delandistrogene Moxeparvovec Gene Therapy in Ambulatory Patients (Aged ≥4 to <8 Years) with Duchenne Muscular Dystrophy: 1-Year Interim Results from Study SRP-9001-103 (ENDEAVOR).
Zaidman CM, Proud CM, McDonald CM, Lehman KJ, Goedeker NL, Mason S, Murphy AP, Guridi M, Wang S, Reid C, Darton E, Wandel C, Lewis S, Malhotra J, Griffin DA, Potter RA, Rodino-Klapac LR, Mendell JR. Zaidman CM, et al. Among authors: potter ra. Ann Neurol. 2023 Nov;94(5):955-968. doi: 10.1002/ana.26755. Epub 2023 Sep 7. Ann Neurol. 2023. PMID: 37539981
Expression of SRP-9001 dystrophin and stabilization of motor function up to 2 years post-treatment with delandistrogene moxeparvovec gene therapy in individuals with Duchenne muscular dystrophy.
Mendell JR, Shieh PB, McDonald CM, Sahenk Z, Lehman KJ, Lowes LP, Reash NF, Iammarino MA, Alfano LN, Sabo B, Woods JD, Skura CL, Mao HC, Staudt LA, Griffin DA, Lewis S, Wang S, Potter RA, Singh T, Rodino-Klapac LR. Mendell JR, et al. Among authors: potter ra. Front Cell Dev Biol. 2023 Jul 11;11:1167762. doi: 10.3389/fcell.2023.1167762. eCollection 2023. Front Cell Dev Biol. 2023. PMID: 37497476 Free PMC article.
Systemic γ-sarcoglycan AAV gene transfer results in dose-dependent correction of muscle deficits in the LGMD 2C/R5 mouse model.
Seo YE, Baine SH, Kempton AN, Rogers OC, Lewis S, Adegboye K, Haile A, Griffin DA, Peterson EL, Pozsgai ER, Potter RA, Rodino-Klapac LR. Seo YE, et al. Among authors: potter ra. Mol Ther Methods Clin Dev. 2023 Jan 16;28:284-299. doi: 10.1016/j.omtm.2023.01.004. eCollection 2023 Mar 9. Mol Ther Methods Clin Dev. 2023. PMID: 36816759 Free PMC article.
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