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Page 1
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: rico s. EBioMedicine. 2024 Jan;99:104894. doi: 10.1016/j.ebiom.2023.104894. Epub 2023 Dec 12. EBioMedicine. 2024. PMID: 38086156 Free PMC article.
Severe communication delays are independent of seizure burden and persist despite contemporary treatments in SCN1A+ Dravet syndrome: Insights from the ENVISION natural history study.
Perry MS, Scheffer IE, Sullivan J, Brunklaus A, Boronat S, Wheless JW, Laux L, Patel AD, Roberts CM, Dlugos D, Holder D, Knupp KG, Lallas M, Phillips S, Segal E, Smeyers P, Lal D, Wirrell E, Zuberi S, Brünger T, Wojnaroski M, Maru B, O'Donnell P, Morton M, James E, Vila MC, Huang N, Gofshteyn JS, Rico S. Perry MS, et al. Among authors: rico s. Epilepsia. 2024 Feb;65(2):322-337. doi: 10.1111/epi.17850. Epub 2023 Dec 22. Epilepsia. 2024. PMID: 38049202 Free 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: rico s. 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.
Influence of vascular imaging acquisition at local stroke centers on workflows in the drip-n-ship model: a RACECAT post hoc analysis.
Flores A, Garcia-Tornel A, Seró L, Ustrell X, Requena M, Pellisé A, Rodriguez P, Monterde A, Lara L, Gonzalez-de-Echavarri JM, Molina CA, Doncel-Moriano A, Dorado L, Cardona P, Cánovas D, Krupinski J, Más N, Purroy F, Zaragoza-Brunet J, Palomeras E, Cocho D, Garcia J, Colom C, Silva Y, Gomez-Cocho M, Jiménez X, Ros-Roig J, Abilleira S, Pérez de la Ossa N, Ribo M; RACECAT trial investigators. Flores A, et al. J Neurointerv Surg. 2024 Jan 12;16(2):143-150. doi: 10.1136/jnis-2023-020125. J Neurointerv Surg. 2024. PMID: 37068936 Clinical Trial.
PD-L1 expression and CD8 positive lymphocytes in human neoplasms: A tissue microarray study on 11,838 tumor samples.
Möller K, Knöll M, Bady E, Schmerder MJ, Rico SD, Kluth M, Hube-Magg C, Blessin NC, Mandelkow T, Lennartz M, Menz A, Luebke AM, Höflmayer D, Fraune C, Bernreuther C, Lebok P, Uhlig R, Contreras H, Weidemann S, Gorbokon N, Jacobsen F, Clauditz TS, Steurer S, Burandt E, Minner S, Sauter G, Simon R, Marx AH, Krech T. Möller K, et al. Among authors: rico sd. Cancer Biomark. 2023;36(2):177-191. doi: 10.3233/CBM-220030. Cancer Biomark. 2023. PMID: 36683495 Free PMC article.
78 results