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A gene therapy targeting medium-chain acyl-CoA dehydrogenase (MCAD) did not protect against diabetes-induced cardiac pathology.
Weeks KL, Kiriazis H, Wadley GD, Masterman EI, Sergienko NM, Raaijmakers AJA, Trewin AJ, Harmawan CA, Yildiz GS, Liu Y, Drew BG, Gregorevic P, Delbridge LMD, McMullen JR, Bernardo BC. Weeks KL, et al. J Mol Med (Berl). 2024 Jan;102(1):95-111. doi: 10.1007/s00109-023-02397-2. Epub 2023 Nov 21. J Mol Med (Berl). 2024. PMID: 37987775
Rapid centralised randomisation in emergency setting trials using a smartphone.
Badurdeen S, Hodgson KA, Santomartino GA, Stevens L, Donath S, Roberts CT, Manley BJ, Polglase GR, Hooper SB, Davis PG, Blank DA. Badurdeen S, et al. Eur J Pediatr. 2022 Aug;181(8):3207-3210. doi: 10.1007/s00431-022-04475-y. Epub 2022 May 17. Eur J Pediatr. 2022. PMID: 35579708 Free PMC article. Clinical Trial.