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The TREAT-NMD advisory committee for therapeutics (TACT): an innovative de-risking model to foster orphan drug development.
Heslop E, Csimma C, Straub V, McCall J, Nagaraju K, Wagner KR, Caizergues D, Korinthenberg R, Flanigan KM, Kaufmann P, McNeil E, Mendell J, Hesterlee S, Wells DJ, Bushby K; TACT. Heslop E, et al. Among authors: straub v. Orphanet J Rare Dis. 2015 Apr 23;10:49. doi: 10.1186/s13023-015-0258-1. Orphanet J Rare Dis. 2015. PMID: 25902795 Free PMC article. Review.
Cardiac and respiratory failure in limb-girdle muscular dystrophy 2I.
Poppe M, Bourke J, Eagle M, Frosk P, Wrogemann K, Greenberg C, Muntoni F, Voit T, Straub V, Hilton-Jones D, Shirodaria C, Bushby K. Poppe M, et al. Among authors: straub v. Ann Neurol. 2004 Nov;56(5):738-41. doi: 10.1002/ana.20283. Ann Neurol. 2004. PMID: 15505776
Nonmolecular treatment for muscular dystrophies.
Bushby K, Straub V. Bushby K, et al. Among authors: straub v. Curr Opin Neurol. 2005 Oct;18(5):511-8. doi: 10.1097/01.wco.0000181326.86292.aa. Curr Opin Neurol. 2005. PMID: 16155433 Review.
Severe phenotype in infantile facioscapulohumeral muscular dystrophy.
Klinge L, Eagle M, Haggerty ID, Roberts CE, Straub V, Bushby KM. Klinge L, et al. Among authors: straub v. Neuromuscul Disord. 2006 Oct;16(9-10):553-8. doi: 10.1016/j.nmd.2006.06.008. Epub 2006 Aug 24. Neuromuscul Disord. 2006. PMID: 16934468
The childhood limb-girdle muscular dystrophies.
Straub V, Bushby K. Straub V, et al. Semin Pediatr Neurol. 2006 Jun;13(2):104-14. doi: 10.1016/j.spen.2006.06.006. Semin Pediatr Neurol. 2006. PMID: 17027860 Review.
The limb-girdle muscular dystrophies--diagnostic strategies.
Bushby K, Norwood F, Straub V. Bushby K, et al. Among authors: straub v. Biochim Biophys Acta. 2007 Feb;1772(2):238-42. doi: 10.1016/j.bbadis.2006.09.009. Epub 2006 Oct 3. Biochim Biophys Acta. 2007. PMID: 17123791 Free article.
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. Among authors: straub v. Ann Neurol. 2008 May;63(5):561-71. doi: 10.1002/ana.21338. Ann Neurol. 2008. PMID: 18335515 Clinical Trial.
494 results