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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: sepulveda b. EBioMedicine. 2024 Jan;99:104894. doi: 10.1016/j.ebiom.2023.104894. Epub 2023 Dec 12. EBioMedicine. 2024. PMID: 38086156 Free PMC article.
INCEPTUS Natural History, Run-in Study for Gene Replacement Clinical Trial in X-Linked Myotubular Myopathy.
Dowling JJ, Müller-Felber W, Smith BK, Bönnemann CG, Kuntz NL, Muntoni F, Servais L, Alfano LN, Beggs AH, Bilder DA, Blaschek A, Duong T, Graham RJ, Jain M, Lawlor MW, Lee J, Coats J, Lilien C, Lowes LP, MacBean V, Neuhaus S, Noursalehi M, Pitts T, Finlay C, Christensen S, Rafferty G, Seferian AM, Tsuchiya E, James ES, Miller W, Sepulveda B, Vila MC, Prasad S, Rico S, Shieh PB; INCEPTUS investigators. Dowling JJ, et al. Among authors: sepulveda b. J Neuromuscul Dis. 2022;9(4):503-516. doi: 10.3233/JND-210781. J Neuromuscul Dis. 2022. PMID: 35694931 Free PMC article.
Intrahepatic Cholestasis Is a Clinically Significant Feature Associated with Natural History of X-Linked Myotubular Myopathy (XLMTM): A Case Series and Biopsy Report.
Molera C, Sarishvili T, Nascimento A, Rtskhiladze I, Muñoz Bartolo G, Fernández Cebrián S, Valverde Fernández J, Muñoz Cabello B, Graham RJ, Miller W, Sepulveda B, Kamath BM, Meng H, Lawlor MW. Molera C, et al. Among authors: sepulveda b. J Neuromuscul Dis. 2022;9(1):73-82. doi: 10.3233/JND-210712. J Neuromuscul Dis. 2022. PMID: 34366366 Free PMC article.
The efficient magneto-mechanical actuation of cancer cells using a very low concentration of non-interacting ferrimagnetic hexaferrite nanoplatelets.
Goršak T, Jovičić EJ, Tratnjek L, Križaj I, Sepulveda B, Nogues J, Kreft ME, Petan T, Kralj S, Makovec D. Goršak T, et al. Among authors: sepulveda b. J Colloid Interface Sci. 2024 Mar;657:778-787. doi: 10.1016/j.jcis.2023.12.019. Epub 2023 Dec 4. J Colloid Interface Sci. 2024. PMID: 38081112 Free article.
Enhanced Proliferation and Differentiation of Human Osteoblasts by Remotely Controlled Magnetic-Field-Induced Electric Stimulation Using Flexible Substrates.
Careta O, Nicolenco A, Perdikos F, Blanquer A, Ibañez E, Pellicer E, Stefani C, Sepúlveda B, Nogués J, Sort J, Nogués C. Careta O, et al. Among authors: sepulveda b. ACS Appl Mater Interfaces. 2023 Dec 20;15(50):58054-58066. doi: 10.1021/acsami.3c09428. Epub 2023 Dec 5. ACS Appl Mater Interfaces. 2023. PMID: 38051712 Free PMC article.
225 results