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Year Number of Results
2003 1
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2009 1
2010 5
2011 4
2012 5
2013 7
2014 7
2015 1
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2021 4
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42 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: sproule dm. 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.
Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy in patients with two copies of SMN2 (STR1VE): an open-label, single-arm, multicentre, phase 3 trial.
Day JW, Finkel RS, Chiriboga CA, Connolly AM, Crawford TO, Darras BT, Iannaccone ST, Kuntz NL, Peña LDM, Shieh PB, Smith EC, Kwon JM, Zaidman CM, Schultz M, Feltner DE, Tauscher-Wisniewski S, Ouyang H, Chand DH, Sproule DM, Macek TA, Mendell JR. Day JW, et al. Among authors: sproule dm. Lancet Neurol. 2021 Apr;20(4):284-293. doi: 10.1016/S1474-4422(21)00001-6. Epub 2021 Mar 17. Lancet Neurol. 2021. PMID: 33743238 Clinical Trial.
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: sproule dm. 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.
Observational study of spinal muscular atrophy type I and implications for clinical trials.
Finkel RS, McDermott MP, Kaufmann P, Darras BT, Chung WK, Sproule DM, Kang PB, Foley AR, Yang ML, Martens WB, Oskoui M, Glanzman AM, Flickinger J, Montes J, Dunaway S, O'Hagen J, Quigley J, Riley S, Benton M, Ryan PA, Montgomery M, Marra J, Gooch C, De Vivo DC. Finkel RS, et al. Among authors: sproule dm. Neurology. 2014 Aug 26;83(9):810-7. doi: 10.1212/WNL.0000000000000741. Epub 2014 Jul 30. Neurology. 2014. PMID: 25080519 Free PMC article.
The 6-minute walk test and other clinical endpoints in duchenne muscular dystrophy: reliability, concurrent validity, and minimal clinically important differences from a multicenter study.
McDonald CM, Henricson EK, Abresch RT, Florence J, Eagle M, Gappmaier E, Glanzman AM; PTC124-GD-007-DMD Study Group; Spiegel R, Barth J, Elfring G, Reha A, Peltz SW. McDonald CM, et al. Muscle Nerve. 2013 Sep;48(3):357-68. doi: 10.1002/mus.23905. Epub 2013 Jul 17. Muscle Nerve. 2013. PMID: 23674289 Free PMC article. Clinical Trial.
Reply: To PMID 23893312.
Kang PB, Gooch CL, McDermott MP, Darras BT, Finkel RS, Yang ML, Sproule DM, Chung WK, Kaufmann P, De Vivo DC; Muscle Study Group and the Pediatric Neuromuscular Clinical Research Network for Spinal Muscular Atrophy. Kang PB, et al. Among authors: sproule dm. Muscle Nerve. 2014 Sep;50(3):458-9. doi: 10.1002/mus.24317. Epub 2014 Aug 5. Muscle Nerve. 2014. PMID: 24935909 No abstract available.
Wolff-Parkinson-White syndrome in Patients With MELAS.
Sproule DM, Kaufmann P, Engelstad K, Starc TJ, Hordof AJ, De Vivo DC. Sproule DM, et al. Arch Neurol. 2007 Nov;64(11):1625-7. doi: 10.1001/archneur.64.11.1625. Arch Neurol. 2007. PMID: 17998445
Weakness and fatigue in diverse neuromuscular diseases.
Montes J, Blumenschine M, Dunaway S, Alter AS, Engelstad K, Rao AK, Chiriboga CA, Sproule DM, De Vivo DC. Montes J, et al. Among authors: sproule dm. J Child Neurol. 2013 Oct;28(10):1277-83. doi: 10.1177/0883073813493663. Epub 2013 Jul 11. J Child Neurol. 2013. PMID: 23847297
42 results