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16 results

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Page 1
Transitioning From Nusinersen to Risdiplam for Spinal Muscular Atrophy in Clinical Practice: A Single-Center Experience.
Bekircan-Kurt CE, Subramanian S, Chagat S, Mackenzie SJ, Iammarino M, Reash N, Richardson C, Tsao CY, Noritz G, Gushue C, Kotha K, Paul G, Shell R, Alfano LN, Lowes LP, Connolly AM, Waldrop MA. Bekircan-Kurt CE, et al. Among authors: reash n. Muscle Nerve. 2025 Jan 2. doi: 10.1002/mus.28329. Online ahead of print. Muscle Nerve. 2025. PMID: 39744899
Prospective observational study of FKRP-related limb-girdle muscular dystrophy R9: A GRASP consortium study.
Alfano LN, James MK, Grosfjeld Petersen K, Rudolf K, Vissing J, Augsburger R, Mozaffar T, Jones A, Butler A, Laubscher KM, Mockler SRH, Mathews KD, Iammarino MA, Reash NF, Pietruszewski L, Lowes LP, Strahler T, Wicklund M, Hunn S, Weihl CC, Sasidharan S, Currence M, Statland JM, Stinson N, Holzer M, Leung DG, Lott DJ, Kang PB, Holsten S, Desai U, Johnson NE; GRASP‐LGMD Consortium. Alfano LN, et al. Among authors: reash nf. Ann Clin Transl Neurol. 2024 Dec 15. doi: 10.1002/acn3.52276. Online ahead of print. Ann Clin Transl Neurol. 2024. PMID: 39675022 Free article.
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.
Continued safety and long-term effectiveness of onasemnogene abeparvovec in Ohio.
Waldrop MA, Chagat S, Storey M, Meyer A, Iammarino M, Reash N, Alfano L, Lowes L, Noritz G, Prochoroff A, Rossman I, Ginsberg M, Mosher K, Broomall E, Bass N, Gushue C, Kotha K, Paul G, Shell R, Tsao CY, Mendell JR, Connolly AM. Waldrop MA, et al. Among authors: reash n. Neuromuscul Disord. 2024 Jan;34:41-48. doi: 10.1016/j.nmd.2023.11.010. Epub 2023 Dec 2. Neuromuscul Disord. 2024. PMID: 38142474
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.
16 results