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Guidance in social and ethical issues related to clinical, diagnostic care and novel therapies for hereditary neuromuscular rare diseases: "translating" the translational.
McCormack P, Woods S, Aartsma-Rus A, Hagger L, Herczegfalvi A, Heslop E, Irwin J, Kirschner J, Moeschen P, Muntoni F, Ouillade MC, Rahbek J, Rehmann-Sutter C, Rouault F, Sejersen T, Vroom E, Straub V, Bushby K, Ferlini A. McCormack P, et al. Among authors: ferlini a. PLoS Curr. 2013 Jan 10;5:ecurrents.md.f90b49429fa814bd26c5b22b13d773ec. doi: 10.1371/currents.md.f90b49429fa814bd26c5b22b13d773ec. PLoS Curr. 2013. PMID: 23330068 Free PMC article.
Biochemical characterization of patients with in-frame or out-of-frame DMD deletions pertinent to exon 44 or 45 skipping.
Anthony K, Arechavala-Gomeza V, Ricotti V, Torelli S, Feng L, Janghra N, Tasca G, Guglieri M, Barresi R, Armaroli A, Ferlini A, Bushby K, Straub V, Ricci E, Sewry C, Morgan J, Muntoni F. Anthony K, et al. Among authors: ferlini a. JAMA Neurol. 2014 Jan;71(1):32-40. doi: 10.1001/jamaneurol.2013.4908. JAMA Neurol. 2014. PMID: 24217213 Free article. Clinical Trial.
Affinity proteomics within rare diseases: a BIO-NMD study for blood biomarkers of muscular dystrophies.
Ayoglu B, Chaouch A, Lochmüller H, Politano L, Bertini E, Spitali P, Hiller M, Niks EH, Gualandi F, Pontén F, Bushby K, Aartsma-Rus A, Schwartz E, Le Priol Y, Straub V, Uhlén M, Cirak S, 't Hoen PA, Muntoni F, Ferlini A, Schwenk JM, Nilsson P, Al-Khalili Szigyarto C. Ayoglu B, et al. Among authors: ferlini a. EMBO Mol Med. 2014 Jul;6(7):918-36. doi: 10.15252/emmm.201303724. EMBO Mol Med. 2014. PMID: 24920607 Free PMC article.
Biomarkers and surrogate endpoints in Duchenne: meeting report.
Aartsma-Rus A, Ferlini A, Vroom E. Aartsma-Rus A, et al. Among authors: ferlini a. Neuromuscul Disord. 2014 Aug;24(8):743-5. doi: 10.1016/j.nmd.2014.03.006. Epub 2014 May 28. Neuromuscul Disord. 2014. PMID: 24951452 No abstract available.
Translational and regulatory challenges for exon skipping therapies.
Aartsma-Rus A, Ferlini A, Goemans N, Pasmooij AM, Wells DJ, Bushby K, Vroom E, Balabanov P. Aartsma-Rus A, et al. Among authors: ferlini a. Hum Gene Ther. 2014 Oct;25(10):885-92. doi: 10.1089/hum.2014.086. Hum Gene Ther. 2014. PMID: 25184444 Review.
Validation of genetic modifiers for Duchenne muscular dystrophy: a multicentre study assessing SPP1 and LTBP4 variants.
van den Bergen JC, Hiller M, Böhringer S, Vijfhuizen L, Ginjaar HB, Chaouch A, Bushby K, Straub V, Scoto M, Cirak S, Humbertclaude V, Claustres M, Scotton C, Passarelli C, Lochmüller H, Muntoni F, Tuffery-Giraud S, Ferlini A, Aartsma-Rus AM, Verschuuren JJ, 't Hoen PA, Spitali P. van den Bergen JC, et al. Among authors: ferlini a. J Neurol Neurosurg Psychiatry. 2015 Oct;86(10):1060-5. doi: 10.1136/jnnp-2014-308409. Epub 2014 Dec 4. J Neurol Neurosurg Psychiatry. 2015. PMID: 25476005 Free PMC article.
317 results