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Page 1
Efficacy and Safety of Vamorolone vs Placebo and Prednisone Among Boys With Duchenne Muscular Dystrophy: A Randomized Clinical Trial.
Guglieri M, Clemens PR, Perlman SJ, Smith EC, Horrocks I, Finkel RS, Mah JK, Deconinck N, Goemans N, Haberlova J, Straub V, Mengle-Gaw LJ, Schwartz BD, Harper AD, Shieh PB, De Waele L, Castro D, Yang ML, Ryan MM, McDonald CM, Tulinius M, Webster R, McMillan HJ, Kuntz NL, Rao VK, Baranello G, Spinty S, Childs AM, Sbrocchi AM, Selby KA, Monduy M, Nevo Y, Vilchez-Padilla JJ, Nascimento-Osorio A, Niks EH, de Groot IJM, Katsalouli M, James MK, van den Anker J, Damsker JM, Ahmet A, Ward LM, Jaros M, Shale P, Dang UJ, Hoffman EP. Guglieri M, et al. Among authors: goemans n. JAMA Neurol. 2022 Oct 1;79(10):1005-1014. doi: 10.1001/jamaneurol.2022.2480. JAMA Neurol. 2022. PMID: 36036925 Free PMC article. Clinical Trial.
CINRG randomized controlled trial of creatine and glutamine in Duchenne muscular dystrophy.
Escolar DM, Buyse G, Henricson E, Leshner R, Florence J, Mayhew J, Tesi-Rocha C, Gorni K, Pasquali L, Patel KM, McCarter R, Huang J, Mayhew T, Bertorini T, Carlo J, Connolly AM, Clemens PR, Goemans N, Iannaccone ST, Igarashi M, Nevo Y, Pestronk A, Subramony SH, Vedanarayanan VV, Wessel H; CINRG Group. Escolar DM, et al. Among authors: goemans n. Ann Neurol. 2005 Jul;58(1):151-5. doi: 10.1002/ana.20523. Ann Neurol. 2005. PMID: 15984021 Clinical Trial.
CINRG pilot trial of oxatomide in steroid-naïve Duchenne muscular dystrophy.
Buyse GM, Goemans N, Henricson E, Jara A, van den Hauwe M, Leshner R, Florence JM, Mayhew JE, Escolar DM. Buyse GM, et al. Among authors: goemans n. Eur J Paediatr Neurol. 2007 Nov;11(6):337-40. doi: 10.1016/j.ejpn.2007.02.009. Epub 2007 Apr 24. Eur J Paediatr Neurol. 2007. PMID: 17459739 Clinical Trial.
Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management.
Bushby K, Finkel R, Birnkrant DJ, Case LE, Clemens PR, Cripe L, Kaul A, Kinnett K, McDonald C, Pandya S, Poysky J, Shapiro F, Tomezsko J, Constantin C; DMD Care Considerations Working Group. Bushby K, et al. Lancet Neurol. 2010 Jan;9(1):77-93. doi: 10.1016/S1474-4422(09)70271-6. Epub 2009 Nov 27. Lancet Neurol. 2010. PMID: 19945913 Review.
Systemic administration of PRO051 in Duchenne's muscular dystrophy.
Goemans NM, Tulinius M, van den Akker JT, Burm BE, Ekhart PF, Heuvelmans N, Holling T, Janson AA, Platenburg GJ, Sipkens JA, Sitsen JM, Aartsma-Rus A, van Ommen GJ, Buyse G, Darin N, Verschuuren JJ, Campion GV, de Kimpe SJ, van Deutekom JC. Goemans NM, et al. N Engl J Med. 2011 Apr 21;364(16):1513-22. doi: 10.1056/NEJMoa1011367. Epub 2011 Mar 23. N Engl J Med. 2011. PMID: 21428760 Free article. Clinical Trial.
International workshop on assessment of upper limb function in Duchenne Muscular Dystrophy: Rome, 15-16 February 2012.
Mercuri E, McDonald C, Mayhew A, Florence J, Mazzone E, Bianco F, Decostre V, Servais L, Ricotti V, Goemans N, Vroom E. Mercuri E, et al. Among authors: goemans n. Neuromuscul Disord. 2012 Nov;22(11):1025-8. doi: 10.1016/j.nmd.2012.06.006. Epub 2012 Jul 13. Neuromuscul Disord. 2012. PMID: 22795657 Free PMC article. No abstract available.
155 results