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Effects of gene replacement therapy with resamirigene bilparvovec (AT132) on skeletal muscle pathology in X-linked myotubular myopathy: results from a substudy of the ASPIRO open-label clinical trial.
Lawlor MW, Schoser B, Margeta M, Sewry CA, Jones KA, Shieh PB, Kuntz NL, Smith BK, Dowling JJ, Müller-Felber W, Bönnemann CG, Seferian AM, Blaschek A, Neuhaus S, Foley AR, Saade DN, Tsuchiya E, Qasim UR, Beatka M, Prom MJ, Ott E, Danielson S, Krakau P, Kumar SN, Meng H, Vanden Avond M, Wells C, Gordish-Dressman H, Beggs AH, Christensen S, Conner E, James ES, Lee J, Sadhu C, Miller W, Sepulveda B, Varfaj F, Prasad S, Rico S. Lawlor MW, et al. Among authors: blaschek a. EBioMedicine. 2024 Jan;99:104894. doi: 10.1016/j.ebiom.2023.104894. Epub 2023 Dec 12. EBioMedicine. 2024. PMID: 38086156 Free PMC article.
Safety and efficacy of gene replacement therapy for X-linked myotubular myopathy (ASPIRO): a multinational, open-label, dose-escalation trial.
Shieh PB, Kuntz NL, Dowling JJ, Müller-Felber W, Bönnemann CG, Seferian AM, Servais L, Smith BK, Muntoni F, Blaschek A, Foley AR, Saade DN, Neuhaus S, Alfano LN, Beggs AH, Buj-Bello A, Childers MK, Duong T, Graham RJ, Jain M, Coats J, MacBean V, James ES, Lee J, Mavilio F, Miller W, Varfaj F, Murtagh M, Han C, Noursalehi M, Lawlor MW, Prasad S, Rico S. Shieh PB, et al. Among authors: blaschek a. Lancet Neurol. 2023 Dec;22(12):1125-1139. doi: 10.1016/S1474-4422(23)00313-7. Lancet Neurol. 2023. PMID: 37977713 Free article. Clinical Trial.
Natalizumab therapy for highly active pediatric multiple sclerosis.
Kornek B, Aboul-Enein F, Rostasy K, Milos RI, Steiner I, Penzien J, Hellwig K, Pitarokoili K, Storm van's Gravesande K, Karenfort M, Blaschek A, Meyer A, Seidl R, Debelic D, Vass K, Prayer D, Kristoferitsch W, Bayas A. Kornek B, et al. Among authors: blaschek a. JAMA Neurol. 2013 Apr;70(4):469-75. doi: 10.1001/jamaneurol.2013.923. JAMA Neurol. 2013. PMID: 23420110
Oligoclonal bands predict multiple sclerosis in children with optic neuritis.
Heussinger N, Kontopantelis E, Gburek-Augustat J, Jenke A, Vollrath G, Korinthenberg R, Hofstetter P, Meyer S, Brecht I, Kornek B, Herkenrath P, Schimmel M, Wenner K, Häusler M, Lutz S, Karenfort M, Blaschek A, Smitka M, Karch S, Piepkorn M, Rostasy K, Lücke T, Weber P, Trollmann R, Klepper J, Häussler M, Hofmann R, Weissert R, Merkenschlager A, Buttmann M; for GRACE-MS (German-speaking Research Alliance for ChildrEn with Multiple Sclerosis). Heussinger N, et al. Among authors: blaschek a. Ann Neurol. 2015 Jun;77(6):1076-82. doi: 10.1002/ana.24409. Epub 2015 May 11. Ann Neurol. 2015. PMID: 25820181
107 results