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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: guglieri m. 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.
Limb-girdle muscular dystrophies.
Guglieri M, Straub V, Bushby K, Lochmüller H. Guglieri M, et al. Curr Opin Neurol. 2008 Oct;21(5):576-84. doi: 10.1097/WCO.0b013e32830efdc2. Curr Opin Neurol. 2008. PMID: 18769252 Review.
Molecular treatments in Duchenne muscular dystrophy.
Guglieri M, Bushby K. Guglieri M, et al. Curr Opin Pharmacol. 2010 Jun;10(3):331-7. doi: 10.1016/j.coph.2010.03.005. Epub 2010 Apr 29. Curr Opin Pharmacol. 2010. PMID: 20434401 Review.
Recessive mutations in RYR1 are a common cause of congenital fiber type disproportion.
Clarke NF, Waddell LB, Cooper ST, Perry M, Smith RL, Kornberg AJ, Muntoni F, Lillis S, Straub V, Bushby K, Guglieri M, King MD, Farrell MA, Marty I, Lunardi J, Monnier N, North KN. Clarke NF, et al. Among authors: guglieri m. Hum Mutat. 2010 Jul;31(7):E1544-50. doi: 10.1002/humu.21278. Hum Mutat. 2010. PMID: 20583297
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: guglieri m. 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.
Neurocognitive profiles in Duchenne muscular dystrophy and gene mutation site.
D'Angelo MG, Lorusso ML, Civati F, Comi GP, Magri F, Del Bo R, Guglieri M, Molteni M, Turconi AC, Bresolin N. D'Angelo MG, et al. Among authors: guglieri m. Pediatr Neurol. 2011 Nov;45(5):292-9. doi: 10.1016/j.pediatrneurol.2011.08.003. Pediatr Neurol. 2011. PMID: 22000308 Free PMC article.
Emotional impact of a paediatric exon-skipping therapy trial.
Garralda ME, Kinali M, Cirak S, Bushby K, Guglieri M, Straub V, Muntoni F. Garralda ME, et al. Among authors: guglieri m. Dev Med Child Neurol. 2011 Dec;53(12):1157-9. doi: 10.1111/j.1469-8749.2011.04128.x. Epub 2011 Nov 8. Dev Med Child Neurol. 2011. PMID: 22066908 Free article. No abstract available.
148 results