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Table representation of search results timeline featuring number of search results per year.

Year Number of Results
1957 1
1995 2
1996 3
1997 4
1998 1
1999 2
2000 2
2001 2
2002 1
2003 1
2004 3
2005 2
2006 2
2008 2
2009 4
2010 3
2011 7
2012 8
2013 8
2014 14
2015 15
2016 13
2017 10
2018 12
2019 10
2020 9
2021 19
2022 10
2023 9
2024 9

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165 results

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Page 1
Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy.
Mendell JR, Al-Zaidy S, Shell R, Arnold WD, Rodino-Klapac LR, Prior TW, Lowes L, Alfano L, Berry K, Church K, Kissel JT, Nagendran S, L'Italien J, Sproule DM, Wells C, Cardenas JA, Heitzer MD, Kaspar A, Corcoran S, Braun L, Likhite S, Miranda C, Meyer K, Foust KD, Burghes AHM, Kaspar BK. Mendell JR, et al. Among authors: lowes l. N Engl J Med. 2017 Nov 2;377(18):1713-1722. doi: 10.1056/NEJMoa1706198. N Engl J Med. 2017. PMID: 29091557 Free article. Clinical Trial.
Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular Dystrophy: A Nonrandomized Controlled Trial.
Mendell JR, Sahenk Z, Lehman K, Nease C, Lowes LP, Miller NF, Iammarino MA, Alfano LN, Nicholl A, Al-Zaidy S, Lewis S, Church K, Shell R, Cripe LH, Potter RA, Griffin DA, Pozsgai E, Dugar A, Hogan M, Rodino-Klapac LR. Mendell JR, et al. Among authors: lowes lp. JAMA Neurol. 2020 Sep 1;77(9):1122-1131. doi: 10.1001/jamaneurol.2020.1484. JAMA Neurol. 2020. PMID: 32539076 Free PMC article. Clinical Trial.
Eteplirsen for the treatment of Duchenne muscular dystrophy.
Mendell JR, Rodino-Klapac LR, Sahenk Z, Roush K, Bird L, Lowes LP, Alfano L, Gomez AM, Lewis S, Kota J, Malik V, Shontz K, Walker CM, Flanigan KM, Corridore M, Kean JR, Allen HD, Shilling C, Melia KR, Sazani P, Saoud JB, Kaye EM; Eteplirsen Study Group. Mendell JR, et al. Among authors: lowes lp. Ann Neurol. 2013 Nov;74(5):637-47. doi: 10.1002/ana.23982. Epub 2013 Sep 10. Ann Neurol. 2013. PMID: 23907995 Free article. 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: lowes lp. 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.
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: lowes lp. Muscle Nerve. 2024 Jan;69(1):93-98. doi: 10.1002/mus.27955. Epub 2023 Aug 14. Muscle Nerve. 2024. PMID: 37577753 Clinical Trial.
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: lowes lp. J Neuromuscul Dis. 2024;11(3):679-685. doi: 10.3233/JND-230107. J Neuromuscul Dis. 2024. PMID: 38461513 Free PMC article.
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: lowes l. Neuromuscul Disord. 2024 Jan;34:41-48. doi: 10.1016/j.nmd.2023.11.010. Epub 2023 Dec 2. Neuromuscul Disord. 2024. PMID: 38142474
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: lowes lp. 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.
Defining Clinical Endpoints in Limb Girdle Muscular Dystrophy: A GRASP-LGMD study.
Doody A, Alfano L, Diaz-Manera J, Lowes L, Mozaffar T, Mathews K, Weihl CC, Wicklund M, Statland J, Johnson NE; GRASP-LGMD Consortium. Doody A, et al. Among authors: lowes l. Res Sq [Preprint]. 2023 Oct 6:rs.3.rs-3370395. doi: 10.21203/rs.3.rs-3370395/v1. Res Sq. 2023. Update in: BMC Neurol. 2024 Mar 15;24(1):96. doi: 10.1186/s12883-024-03588-1. PMID: 37886601 Free PMC article. Updated. Preprint.
165 results