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219 results

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Page 1
Assessing Bulbar Function in Spinal Muscular Atrophy Using Patient-Reported Outcomes.
Dunaway Young S, Pasternak A, Duong T, McGrattan KE, Stranberg S, Maczek E, Dias C, Tang W, Parker D, Levine A, Rohan A, Wolford C, Martens W, McDermott MP, Darras BT, Day JW. Dunaway Young S, et al. Among authors: martens w. J Neuromuscul Dis. 2023;10(2):199-209. doi: 10.3233/JND-221573. J Neuromuscul Dis. 2023. PMID: 36776075 Free PMC article.
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. 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
Open-label trial of recombinant human insulin-like growth factor 1/recombinant human insulin-like growth factor binding protein 3 in myotonic dystrophy type 1.
Heatwole CR, Eichinger KJ, Friedman DI, Hilbert JE, Jackson CE, Logigian EL, Martens WB, McDermott MP, Pandya SK, Quinn C, Smirnow AM, Thornton CA, Moxley RT 3rd. Heatwole CR, et al. Arch Neurol. 2011 Jan;68(1):37-44. doi: 10.1001/archneurol.2010.227. Epub 2010 Sep 13. Arch Neurol. 2011. PMID: 20837825 Free PMC article. Clinical Trial.
Validation of the Expanded Hammersmith Functional Motor Scale in spinal muscular atrophy type II and III.
Glanzman AM, O'Hagen JM, McDermott MP, Martens WB, Flickinger J, Riley S, Quigley J, Montes J, Dunaway S, Deng L, Chung WK, Tawil R, Darras BT, De Vivo DC, Kaufmann P, Finkel RS; Pediatric Neuromuscular Clinical Research Network for Spinal Muscular Atrophy (PNCR); Muscle Study Group (MSG). Glanzman AM, et al. J Child Neurol. 2011 Dec;26(12):1499-507. doi: 10.1177/0883073811420294. Epub 2011 Sep 21. J Child Neurol. 2011. PMID: 21940700
Validation of the Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND).
Glanzman AM, McDermott MP, Montes J, Martens WB, Flickinger J, Riley S, Quigley J, Dunaway S, O'Hagen J, Deng L, Chung WK, Tawil R, Darras BT, Yang M, Sproule D, De Vivo DC, Kaufmann P, Finkel RS; Pediatric Neuromuscular Clinical Research Network for Spinal Muscular Atrophy (PNCR); Muscle Study Group (MSG). Glanzman AM, et al. Pediatr Phys Ther. 2011 Winter;23(4):322-6. doi: 10.1097/PEP.0b013e3182351f04. Pediatr Phys Ther. 2011. PMID: 22090068
If you build a rare disease registry, will they enroll and will they use it? Methods and data from the National Registry of Myotonic Dystrophy (DM) and Facioscapulohumeral Muscular Dystrophy (FSHD).
Hilbert JE, Kissel JT, Luebbe EA, Martens WB, McDermott MP, Sanders DB, Tawil R, Thornton CA, Moxley RT 3rd; Registry Scientific Advisory Committee. Hilbert JE, et al. Contemp Clin Trials. 2012 Mar;33(2):302-11. doi: 10.1016/j.cct.2011.11.016. Epub 2011 Nov 26. Contemp Clin Trials. 2012. PMID: 22155025 Free PMC article.
Patient-reported impact of symptoms in myotonic dystrophy type 1 (PRISM-1).
Heatwole C, Bode R, Johnson N, Quinn C, Martens W, McDermott MP, Rothrock N, Thornton C, Vickrey B, Victorson D, Moxley R 3rd. Heatwole C, et al. Among authors: martens w. Neurology. 2012 Jul 24;79(4):348-57. doi: 10.1212/WNL.0b013e318260cbe6. Epub 2012 Jul 11. Neurology. 2012. PMID: 22786587 Free PMC article.
Prospective cohort study of spinal muscular atrophy types 2 and 3.
Kaufmann P, McDermott MP, Darras BT, Finkel RS, Sproule DM, Kang PB, Oskoui M, Constantinescu A, Gooch CL, Foley AR, Yang ML, Tawil R, Chung WK, Martens WB, Montes J, Battista V, O'Hagen J, Dunaway S, Flickinger J, Quigley J, Riley S, Glanzman AM, Benton M, Ryan PA, Punyanitya M, Montgomery MJ, Marra J, Koo B, De Vivo DC; Muscle Study Group (MSG); Pediatric Neuromuscular Clinical Research Network for Spinal Muscular Atrophy (PNCR). Kaufmann P, et al. Neurology. 2012 Oct 30;79(18):1889-97. doi: 10.1212/WNL.0b013e318271f7e4. Epub 2012 Oct 17. Neurology. 2012. PMID: 23077013 Free PMC article.
219 results