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Phase 1 clinical trial of losmapimod in facioscapulohumeral dystrophy: Safety, tolerability, pharmacokinetics, and target engagement.
Mellion ML, Ronco L, Berends CL, Pagan L, Brooks S, van Esdonk MJ, van Brummelen EMJ, Odueyungbo A, Thompson LA, Hage M, Badrising UA, Raines S, Tracewell WG, van Engelen B, Cadavid D, Groeneveld GJ. Mellion ML, et al. Among authors: ronco l. Br J Clin Pharmacol. 2021 Dec;87(12):4658-4669. doi: 10.1111/bcp.14884. Epub 2021 May 14. Br J Clin Pharmacol. 2021. PMID: 33931884 Free article. Clinical Trial.
p38α Regulates Expression of DUX4 in a Model of Facioscapulohumeral Muscular Dystrophy.
Rojas LA, Valentine E, Accorsi A, Maglio J, Shen N, Robertson A, Kazmirski S, Rahl P, Tawil R, Cadavid D, Thompson LA, Ronco L, Chang AN, Cacace AM, Wallace O. Rojas LA, et al. Among authors: ronco l. J Pharmacol Exp Ther. 2020 Sep;374(3):489-498. doi: 10.1124/jpet.119.264689. Epub 2020 Jun 23. J Pharmacol Exp Ther. 2020. PMID: 32576599
Quantitative muscle analysis in facioscapulohumeral muscular dystrophy using whole-body fat-referenced MRI: Protocol development, multicenter feasibility, and repeatability.
Widholm P, Ahlgren A, Karlsson M, Romu T, Tawil R, Wagner KR, Statland JM, Wang LH, Shieh PB, van Engelen BGM, Cadavid D, Ronco L, Odueyungbo AO, Jiang JG, Mellion ML, Dahlqvist Leinhard O. Widholm P, et al. Among authors: ronco l. Muscle Nerve. 2022 Aug;66(2):183-192. doi: 10.1002/mus.27638. Epub 2022 Jun 11. Muscle Nerve. 2022. PMID: 35585766
Quantitative Muscle Analysis in FSHD Using Whole-Body Fat-Referenced MRI: Composite Scores for Longitudinal and Cross-sectional Analysis.
Mellion ML, Widholm P, Karlsson M, Ahlgren A, Tawil R, Wagner KR, Statland JM, Wang L, Shieh PB, van Engelen BGM, Kools J, Ronco L, Odueyungbo A, Jiang J, Han JJ, Hatch M, Towles J, Leinhard OD, Cadavid D. Mellion ML, et al. Among authors: ronco l. Neurology. 2022 Aug 30;99(9):e877-e889. doi: 10.1212/WNL.0000000000200757. Epub 2022 Jun 24. Neurology. 2022. PMID: 35750498
Small Molecule Targets TMED9 and Promotes Lysosomal Degradation to Reverse Proteinopathy.
Dvela-Levitt M, Kost-Alimova M, Emani M, Kohnert E, Thompson R, Sidhom EH, Rivadeneira A, Sahakian N, Roignot J, Papagregoriou G, Montesinos MS, Clark AR, McKinney D, Gutierrez J, Roth M, Ronco L, Elonga E, Carter TA, Gnirke A, Melanson M, Hartland K, Wieder N, Hsu JC, Deltas C, Hughey R, Bleyer AJ, Kmoch S, Živná M, Barešova V, Kota S, Schlondorff J, Heiman M, Alper SL, Wagner F, Weins A, Golub TR, Lander ES, Greka A. Dvela-Levitt M, et al. Among authors: ronco l. Cell. 2019 Jul 25;178(3):521-535.e23. doi: 10.1016/j.cell.2019.07.002. Cell. 2019. PMID: 31348885 Free article.
A small molecule that binds and inhibits the ETV1 transcription factor oncoprotein.
Pop MS, Stransky N, Garvie CW, Theurillat JP, Hartman EC, Lewis TA, Zhong C, Culyba EK, Lin F, Daniels DS, Pagliarini R, Ronco L, Koehler AN, Garraway LA. Pop MS, et al. Among authors: ronco l. Mol Cancer Ther. 2014 Jun;13(6):1492-502. doi: 10.1158/1535-7163.MCT-13-0689. Epub 2014 Apr 15. Mol Cancer Ther. 2014. PMID: 24737027 Free PMC article.
A High Content Screen for Mucin-1-Reducing Compounds Identifies Fostamatinib as a Candidate for Rapid Repurposing for Acute Lung Injury during the COVID-19 pandemic.
Alimova M, Sidhom EH, Satyam A, Dvela-Levitt M, Melanson M, Chamberlain BT, Alper SL, Santos J, Gutierrez J, Subramanian A, Grinkevich E, Bricio ER, Kim C, Clark A, Watts A, Thompson R, Marshall J, Pablo JL, Coraor J, Roignot J, Vernon KA, Keller K, Campbell A, Emani M, Racette M, Bazua-Valenti S, Padovano V, Weins A, McAdoo SP, Tam FWK, Ronco L, Wagner F, Tsokos GC, Shaw JL, Greka A. Alimova M, et al. Among authors: ronco l. bioRxiv [Preprint]. 2020 Jun 30:2020.06.30.180380. doi: 10.1101/2020.06.30.180380. bioRxiv. 2020. PMID: 32637960 Free PMC article. Updated. Preprint.
41 results