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Placebo-controlled crossover assessment of mecasermin for the treatment of Rett syndrome.
O'Leary HM, Kaufmann WE, Barnes KV, Rakesh K, Kapur K, Tarquinio DC, Cantwell NG, Roche KJ, Rose SA, Walco AC, Bruck NM, Bazin GA, Holm IA, Alexander ME, Swanson LC, Baczewski LM, Poon C, Mayor Torres JM, Nelson CA 3rd, Sahin M. O'Leary HM, et al. Among authors: kaufmann we. Ann Clin Transl Neurol. 2018 Jan 31;5(3):323-332. doi: 10.1002/acn3.533. eCollection 2018 Mar. Ann Clin Transl Neurol. 2018. PMID: 29560377 Free PMC article.
Safety, pharmacokinetics, and preliminary assessment of efficacy of mecasermin (recombinant human IGF-1) for the treatment of Rett syndrome.
Khwaja OS, Ho E, Barnes KV, O'Leary HM, Pereira LM, Finkelstein Y, Nelson CA 3rd, Vogel-Farley V, DeGregorio G, Holm IA, Khatwa U, Kapur K, Alexander ME, Finnegan DM, Cantwell NG, Walco AC, Rappaport L, Gregas M, Fichorova RN, Shannon MW, Sur M, Kaufmann WE. Khwaja OS, et al. Among authors: kaufmann we. Proc Natl Acad Sci U S A. 2014 Mar 25;111(12):4596-601. doi: 10.1073/pnas.1311141111. Epub 2014 Mar 12. Proc Natl Acad Sci U S A. 2014. PMID: 24623853 Free PMC article.
Developmental delay in Rett syndrome: data from the natural history study.
Neul JL, Lane JB, Lee HS, Geerts S, Barrish JO, Annese F, Baggett LM, Barnes K, Skinner SA, Motil KJ, Glaze DG, Kaufmann WE, Percy AK. Neul JL, et al. Among authors: kaufmann we. J Neurodev Disord. 2014;6(1):20. doi: 10.1186/1866-1955-6-20. Epub 2014 Jul 22. J Neurodev Disord. 2014. PMID: 25071871 Free PMC article.
Pubertal development in Rett syndrome deviates from typical females.
Killian JT, Lane JB, Cutter GR, Skinner SA, Kaufmann WE, Tarquinio DC, Glaze DG, Motil KJ, Neul JL, Percy AK. Killian JT, et al. Among authors: kaufmann we. Pediatr Neurol. 2014 Dec;51(6):769-75. doi: 10.1016/j.pediatrneurol.2014.08.013. Epub 2014 Aug 29. Pediatr Neurol. 2014. PMID: 25283752 Free PMC article. Clinical Trial.
Age of diagnosis in Rett syndrome: patterns of recognition among diagnosticians and risk factors for late diagnosis.
Tarquinio DC, Hou W, Neul JL, Lane JB, Barnes KV, O'Leary HM, Bruck NM, Kaufmann WE, Motil KJ, Glaze DG, Skinner SA, Annese F, Baggett L, Barrish JO, Geerts SP, Percy AK. Tarquinio DC, et al. Among authors: kaufmann we. Pediatr Neurol. 2015 Jun;52(6):585-91.e2. doi: 10.1016/j.pediatrneurol.2015.02.007. Epub 2015 Feb 16. Pediatr Neurol. 2015. PMID: 25801175 Free PMC article.
The Changing Face of Survival in Rett Syndrome and MECP2-Related Disorders.
Tarquinio DC, Hou W, Neul JL, Kaufmann WE, Glaze DG, Motil KJ, Skinner SA, Lee HS, Percy AK. Tarquinio DC, et al. Among authors: kaufmann we. Pediatr Neurol. 2015 Nov;53(5):402-11. doi: 10.1016/j.pediatrneurol.2015.06.003. Epub 2015 Jun 26. Pediatr Neurol. 2015. PMID: 26278631 Free PMC article.
Visual evoked potentials detect cortical processing deficits in Rett syndrome.
LeBlanc JJ, DeGregorio G, Centofante E, Vogel-Farley VK, Barnes K, Kaufmann WE, Fagiolini M, Nelson CA. LeBlanc JJ, et al. Among authors: kaufmann we. Ann Neurol. 2015 Nov;78(5):775-86. doi: 10.1002/ana.24513. Epub 2015 Sep 18. Ann Neurol. 2015. PMID: 26332183 Free PMC article.
Detecting autonomic response to pain in Rett syndrome.
O'Leary HM, Marschik PB, Khwaja OS, Ho E, Barnes KV, Clarkson TW, Bruck NM, Kaufmann WE. O'Leary HM, et al. Among authors: kaufmann we. Dev Neurorehabil. 2017 Feb;20(2):108-114. doi: 10.3109/17518423.2015.1087437. Epub 2015 Oct 12. Dev Neurorehabil. 2017. PMID: 26457613
216 results