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Page 1
Increase in Full-Length Dystrophin by Exon Skipping in Duchenne Muscular Dystrophy Patients with Single Exon Duplications: An Open-label Study.
Nicolau S, Malhotra J, Kaler M, Magistrado-Coxen P, Iammarino MA, Reash NF, Frair EC, Wijeratne S, Kelly BJ, White P, Lowes LP, Waldrop MA, Flanigan KM. Nicolau S, et al. Among authors: reash nf. J Neuromuscul Dis. 2024;11(3):679-685. doi: 10.3233/JND-230107. J Neuromuscul Dis. 2024. PMID: 38461513 Free PMC article.
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: reash nf. 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: reash nf. Muscle Nerve. 2024 Jan;69(1):93-98. doi: 10.1002/mus.27955. Epub 2023 Aug 14. Muscle Nerve. 2024. PMID: 37577753 Clinical Trial.
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: reash nf. 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.
Validation of the North Star Assessment for Limb-Girdle Type Muscular Dystrophies.
James MK, Alfano LN, Muni-Lofra R, Reash NF, Sodhi J, Iammarino MA, Moat D, Shannon K, McCallum M, Richardson M, Eagle M, Straub V, Marini-Bettolo C, Lowes LP, Mayhew AG. James MK, et al. Among authors: reash nf. Phys Ther. 2022 Oct 6;102(10):pzac113. doi: 10.1093/ptj/pzac113. Phys Ther. 2022. PMID: 35932452 Free PMC article.
Comparison of strength testing modalities in dysferlinopathy.
Reash NF, James MK, Alfano LN, Mayhew AG, Jacobs M, Iammarino MA, Holsten S, Sakamoto C, Tateishi T, Yajima H, Duong T, de Wolf B, Gee R, Bharucha-Goebel DX, Bravver E, Mori-Yoshimura M, Bushby K, Rufibach LE, Straub V, Lowes LP; Jain COS Consortium. Reash NF, et al. Muscle Nerve. 2022 Aug;66(2):159-166. doi: 10.1002/mus.27570. Epub 2022 May 20. Muscle Nerve. 2022. PMID: 35506767
Validity and Reliability of the Neuromuscular Gross Motor Outcome.
Alfano LN, Iammarino MA, Reash NF, Powers BR, Shannon K, Connolly AM, Waldrop MA, Noritz GH, Shell R, Tsao CY, Flanigan KM, Mendell JR, Lowes LP. Alfano LN, et al. Among authors: reash nf. Pediatr Neurol. 2021 Sep;122:21-26. doi: 10.1016/j.pediatrneurol.2021.05.021. Epub 2021 Jun 8. Pediatr Neurol. 2021. PMID: 34271497
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