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662 results

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Page 1
Targeting RNA to treat neuromuscular disease.
Muntoni F, Wood MJ. Muntoni F, et al. Among authors: wood mj. Nat Rev Drug Discov. 2011 Aug 1;10(8):621-37. doi: 10.1038/nrd3459. Nat Rev Drug Discov. 2011. PMID: 21804598 Review.
Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study.
Kinali M, Arechavala-Gomeza V, Feng L, Cirak S, Hunt D, Adkin C, Guglieri M, Ashton E, Abbs S, Nihoyannopoulos P, Garralda ME, Rutherford M, McCulley C, Popplewell L, Graham IR, Dickson G, Wood MJ, Wells DJ, Wilton SD, Kole R, Straub V, Bushby K, Sewry C, Morgan JE, Muntoni F. Kinali M, et al. Among authors: wood mj. Lancet Neurol. 2009 Oct;8(10):918-28. doi: 10.1016/S1474-4422(09)70211-X. Epub 2009 Aug 25. Lancet Neurol. 2009. PMID: 19713152 Free PMC article. Clinical Trial.
Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study.
Cirak S, Arechavala-Gomeza V, Guglieri M, Feng L, Torelli S, Anthony K, Abbs S, Garralda ME, Bourke J, Wells DJ, Dickson G, Wood MJ, Wilton SD, Straub V, Kole R, Shrewsbury SB, Sewry C, Morgan JE, Bushby K, Muntoni F. Cirak S, et al. Among authors: wood mj. Lancet. 2011 Aug 13;378(9791):595-605. doi: 10.1016/S0140-6736(11)60756-3. Epub 2011 Jul 23. Lancet. 2011. PMID: 21784508 Free PMC article. Clinical Trial.
Splicing therapy for neuromuscular disease.
Douglas AG, Wood MJ. Douglas AG, et al. Among authors: wood mj. Mol Cell Neurosci. 2013 Sep;56:169-85. doi: 10.1016/j.mcn.2013.04.005. Epub 2013 Apr 28. Mol Cell Neurosci. 2013. PMID: 23631896 Free PMC article. Review.
Stakeholder cooperation to overcome challenges in orphan medicine development: the example of Duchenne muscular dystrophy.
Straub V, Balabanov P, Bushby K, Ensini M, Goemans N, De Luca A, Pereda A, Hemmings R, Campion G, Kaye E, Arechavala-Gomeza V, Goyenvalle A, Niks E, Veldhuizen O, Furlong P, Stoyanova-Beninska V, Wood MJ, Johnson A, Mercuri E, Muntoni F, Sepodes B, Haas M, Vroom E, Aartsma-Rus A. Straub V, et al. Among authors: wood mj. Lancet Neurol. 2016 Jul;15(8):882-890. doi: 10.1016/S1474-4422(16)30035-7. Lancet Neurol. 2016. PMID: 27302365 Review.
662 results