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Randomized phase 2 study of ACE-083, a muscle-promoting agent, in facioscapulohumeral muscular dystrophy.
Statland JM, Campbell C, Desai U, Karam C, Díaz-Manera J, Guptill JT, Korngut L, Genge A, Tawil RN, Elman L, Joyce NC, Wagner KR, Manousakis G, Amato AA, Butterfield RJ, Shieh PB, Wicklund M, Gamez J, Bodkin C, Pestronk A, Weihl CC, Vilchez-Padilla JJ, Johnson NE, Mathews KD, Miller B, Leneus A, Fowler M, van de Rijn M, Attie KM. Statland JM, et al. Among authors: tawil rn. Muscle Nerve. 2022 Jul;66(1):50-62. doi: 10.1002/mus.27558. Epub 2022 May 9. Muscle Nerve. 2022. PMID: 35428982 Free PMC article. Clinical Trial.
Facioscapulohumeral muscular dystrophy.
Tawil R. Tawil R. Curr Neurol Neurosci Rep. 2004 Jan;4(1):51-4. doi: 10.1007/s11910-004-0012-x. Curr Neurol Neurosci Rep. 2004. PMID: 14683629 Review.
Facioscapulohumeral muscular dystrophy.
Tawil R, Van Der Maarel SM. Tawil R, et al. Muscle Nerve. 2006 Jul;34(1):1-15. doi: 10.1002/mus.20522. Muscle Nerve. 2006. PMID: 16508966 Review.
A phase I/IItrial of MYO-029 in adult subjects with muscular dystrophy.
Wagner KR, Fleckenstein JL, Amato AA, Barohn RJ, Bushby K, Escolar DM, Flanigan KM, Pestronk A, Tawil R, Wolfe GI, Eagle M, Florence JM, King WM, Pandya S, Straub V, Juneau P, Meyers K, Csimma C, Araujo T, Allen R, Parsons SA, Wozney JM, Lavallie ER, Mendell JR. Wagner KR, et al. Ann Neurol. 2008 May;63(5):561-71. doi: 10.1002/ana.21338. Ann Neurol. 2008. PMID: 18335515 Clinical Trial.
Facioscapulohumeral muscular dystrophy.
Tawil R. Tawil R. Neurotherapeutics. 2008 Oct;5(4):601-6. doi: 10.1016/j.nurt.2008.07.005. Neurotherapeutics. 2008. PMID: 19019312 Free PMC article. Review.
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.
202 results