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Nusinersen initiated in infants during the presymptomatic stage of spinal muscular atrophy: Interim efficacy and safety results from the Phase 2 NURTURE study.
De Vivo DC, Bertini E, Swoboda KJ, Hwu WL, Crawford TO, Finkel RS, Kirschner J, Kuntz NL, Parsons JA, Ryan MM, Butterfield RJ, Topaloglu H, Ben-Omran T, Sansone VA, Jong YJ, Shu F, Staropoli JF, Kerr D, Sandrock AW, Stebbins C, Petrillo M, Braley G, Johnson K, Foster R, Gheuens S, Bhan I, Reyna SP, Fradette S, Farwell W; NURTURE Study Group. De Vivo DC, et al. Neuromuscul Disord. 2019 Nov;29(11):842-856. doi: 10.1016/j.nmd.2019.09.007. Epub 2019 Sep 12. Neuromuscul Disord. 2019. PMID: 31704158 Free PMC article. Clinical Trial.
An expanded version of the Hammersmith Functional Motor Scale for SMA II and III patients.
O'Hagen JM, Glanzman AM, McDermott MP, Ryan PA, Flickinger J, Quigley J, Riley S, Sanborn E, Irvine C, Martens WB, Annis C, Tawil R, Oskoui M, Darras BT, Finkel RS, De Vivo DC. O'Hagen JM, et al. Among authors: de vivo dc. Neuromuscul Disord. 2007 Oct;17(9-10):693-7. doi: 10.1016/j.nmd.2007.05.009. Epub 2007 Jul 19. Neuromuscul Disord. 2007. PMID: 17658255
Clinical outcome measures in spinal muscular atrophy.
Montes J, Gordon AM, Pandya S, De Vivo DC, Kaufmann P. Montes J, et al. Among authors: de vivo dc. J Child Neurol. 2009 Aug;24(8):968-78. doi: 10.1177/0883073809332702. Epub 2009 Jun 9. J Child Neurol. 2009. PMID: 19509409 Review.
Adiposity is increased among high-functioning, non-ambulatory patients with spinal muscular atrophy.
Sproule DM, Montes J, Dunaway S, Montgomery M, Battista V, Koenigsberger D, Martens B, Shen W, Punyanitya M, Benton M, Butler H, Caracciolo J, Mercuri E, Finkel R, Darras B, De Vivo DC, Kaufmann P. Sproule DM, et al. Among authors: de vivo dc. Neuromuscul Disord. 2010 Jul;20(7):448-52. doi: 10.1016/j.nmd.2010.05.013. Epub 2010 Jun 17. Neuromuscul Disord. 2010. PMID: 20610154 Free PMC article.
Muscle volume estimation by magnetic resonance imaging in spinal muscular atrophy.
Sproule DM, Punyanitya M, Shen W, Dashnaw S, Martens B, Montgomery M, Montes J, Battista V, Finkel R, Darras B, De Vivo DC, Kaufmann P. Sproule DM, et al. Among authors: de vivo dc. J Child Neurol. 2011 Mar;26(3):309-17. doi: 10.1177/0883073810380457. Epub 2010 Oct 6. J Child Neurol. 2011. PMID: 20929908
Observational study of spinal muscular atrophy type 2 and 3: functional outcomes over 1 year.
Kaufmann P, McDermott MP, Darras BT, Finkel R, Kang P, Oskoui M, Constantinescu A, Sproule DM, Foley AR, Yang M, Tawil R, Chung W, Martens B, Montes J, O'Hagen J, Dunaway S, Flickinger JM, Quigley J, Riley S, Glanzman AM, Benton M, Ryan PA, Irvine C, Annis CL, Butler H, Caracciolo J, Montgomery M, Marra J, Koo B, De Vivo DC; Muscle Study Group; Pediatric Neuromuscular Clinical Research Network for Spinal Muscular Atrophy. Kaufmann P, et al. Among authors: de vivo dc. Arch Neurol. 2011 Jun;68(6):779-86. doi: 10.1001/archneurol.2010.373. Epub 2011 Feb 14. Arch Neurol. 2011. PMID: 21320981 Free PMC article.
348 results