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

Year Number of Results
2007 2
2008 2
2009 2
2011 1
2012 2
2013 5
2014 4
2015 8
2016 3
2017 5
2018 4
2019 3
2020 9
2021 12
2022 11
2023 3
2024 5

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67 results

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Page 1
Biodistribution of onasemnogene abeparvovec DNA, mRNA and SMN protein in human tissue.
Thomsen G, Burghes AHM, Hsieh C, Do J, Chu BTT, Perry S, Barkho B, Kaufmann P, Sproule DM, Feltner DE, Chung WK, McGovern VL, Hevner RF, Conces M, Pierson CR, Scoto M, Muntoni F, Mendell JR, Foust KD. Thomsen G, et al. Among authors: scoto m. Nat Med. 2021 Oct;27(10):1701-1711. doi: 10.1038/s41591-021-01483-7. Epub 2021 Oct 4. Nat Med. 2021. PMID: 34608334 Clinical Trial.
Revised upper limb module for spinal muscular atrophy: Development of a new module.
Mazzone ES, Mayhew A, Montes J, Ramsey D, Fanelli L, Young SD, Salazar R, De Sanctis R, Pasternak A, Glanzman A, Coratti G, Civitello M, Forcina N, Gee R, Duong T, Pane M, Scoto M, Pera MC, Messina S, Tennekoon G, Day JW, Darras BT, De Vivo DC, Finkel R, Muntoni F, Mercuri E. Mazzone ES, et al. Among authors: scoto m. Muscle Nerve. 2017 Jun;55(6):869-874. doi: 10.1002/mus.25430. Epub 2017 Feb 6. Muscle Nerve. 2017. PMID: 27701745
Long-Term Safety and Efficacy Data of Golodirsen in Ambulatory Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A First-in-human, Multicenter, Two-Part, Open-Label, Phase 1/2 Trial.
Servais L, Mercuri E, Straub V, Guglieri M, Seferian AM, Scoto M, Leone D, Koenig E, Khan N, Dugar A, Wang X, Han B, Wang D, Muntoni F; SKIP-NMD Study Group. Servais L, et al. Among authors: scoto m. Nucleic Acid Ther. 2022 Feb;32(1):29-39. doi: 10.1089/nat.2021.0043. Epub 2021 Nov 17. Nucleic Acid Ther. 2022. PMID: 34788571 Free PMC article. Clinical Trial.
Genetic therapies for inherited neuromuscular disorders.
Scoto M, Finkel R, Mercuri E, Muntoni F. Scoto M, et al. Lancet Child Adolesc Health. 2018 Aug;2(8):600-609. doi: 10.1016/S2352-4642(18)30140-8. Epub 2018 Jun 27. Lancet Child Adolesc Health. 2018. PMID: 30119719 Review.
Dystrophinopathies and Limb-Girdle Muscular Dystrophies.
Domingos J, Sarkozy A, Scoto M, Muntoni F. Domingos J, et al. Among authors: scoto m. Neuropediatrics. 2017 Aug;48(4):262-272. doi: 10.1055/s-0037-1601860. Epub 2017 Apr 20. Neuropediatrics. 2017. PMID: 28427100 Review.
Long-term Natural History of Pediatric Dominant and Recessive RYR1-Related Myopathy.
Sarkozy A, Sa M, Ridout D, Fernandez-Garcia MA, Distefano MG, Main M, Sheehan J, Manzur AY, Munot P, Robb S, Wraige E, Quinlivan R, Scoto M, Baranello G, Gowda V, Mein R, Phadke R, Jungbluth H, Muntoni F. Sarkozy A, et al. Among authors: scoto m. Neurology. 2023 Oct 10;101(15):e1495-e1508. doi: 10.1212/WNL.0000000000207723. Epub 2023 Aug 29. Neurology. 2023. PMID: 37643885
Cardiac Manifestations of Myotonic Dystrophy in a Pediatric Cohort.
Brunet Garcia L, Hajra A, Field E, Wacher J, Walsh H, Norrish G, Manzur A, Muntoni F, Munot P, Robb S, Quinlivan R, Scoto M, Baranello G, Sarkozy A, Starling L, Kaski JP, Cervi E. Brunet Garcia L, et al. Among authors: scoto m. Front Pediatr. 2022 Jun 9;10:910660. doi: 10.3389/fped.2022.910660. eCollection 2022. Front Pediatr. 2022. PMID: 35757141 Free PMC article.
67 results