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Long-term safety and efficacy of cipaglucosidase alfa plus miglustat in individuals living with Pompe disease: an open-label phase I/II study (ATB200-02).
Byrne BJ, Schoser B, Kishnani PS, Bratkovic D, Clemens PR, Goker-Alpan O, Ming X, Roberts M, Vorgerd M, Sivakumar K, van der Ploeg AT, Goldman M, Wright J, Holdbrook F, Jain V, Benjamin ER, Johnson F, Das SS, Wasfi Y, Mozaffar T. Byrne BJ, et al. Among authors: roberts m. J Neurol. 2024 Apr;271(4):1787-1801. doi: 10.1007/s00415-023-12096-0. Epub 2023 Dec 6. J Neurol. 2024. PMID: 38057636 Free PMC article. Clinical Trial.
Switching treatment to cipaglucosidase alfa plus miglustat positively affects patient-reported outcome measures in patients with late-onset Pompe disease.
Kishnani PS, Byrne BJ, Claeys KG, Díaz-Manera J, Dimachkie MM, Kushlaf H, Mozaffar T, Roberts M, Schoser B, Hummel N, Kopiec A, Holdbrook F, Shohet S, Toscano A; PROPEL Study Group. Kishnani PS, et al. Among authors: roberts m. J Patient Rep Outcomes. 2024 Nov 13;8(1):132. doi: 10.1186/s41687-024-00805-w. J Patient Rep Outcomes. 2024. PMID: 39535661 Free PMC article. Clinical Trial.
Effects of leg immobilization and recovery resistance training on skeletal muscle-molecular markers in previously resistance trained versus untrained adults.
Michel JM, Godwin JS, Plotkin DL, McIntosh MC, Mattingly ML, Agostinelli PJ, Mueller BJ, Anglin DA, Kontos NJ, Berry AC, Vega MM, Pipkin AA, Stock MS, Graham ZA, Baweja HS, Mobley CB, Bamman MM, Roberts MD. Michel JM, et al. Among authors: roberts md. J Appl Physiol (1985). 2025 Jan 16. doi: 10.1152/japplphysiol.00837.2024. Online ahead of print. J Appl Physiol (1985). 2025. PMID: 39819075
Baseline Characteristics of Frailty and Disease Stage in Older People Living With CKD.
Logan B, Pascoe EM, Viecelli AK, Johnson DW, Comans T, Hawley CM, Hickey LE, Janda M, Jaure A, Kalaw E, Kiriwandeniya C, Matsuyama M, Mihala G, Nguyen KH, Pole JD, Polkinghorne KR, Pond D, Raj R, Reidlinger DM, Scholes-Robertson N, Valks A, Wong G, Hubbard RE; GOAL Trial Investigators. Logan B, et al. Kidney Int Rep. 2024 Oct 15;10(1):120-133. doi: 10.1016/j.ekir.2024.10.009. eCollection 2025 Jan. Kidney Int Rep. 2024. PMID: 39810773 Free PMC article.
Dual-targeting CRISPR-CasRx reduces C9orf72 ALS/FTD sense and antisense repeat RNAs in vitro and in vivo.
Kempthorne L, Vaizoglu D, Cammack AJ, Carcolé M, Roberts MJ, Mikheenko A, Fisher A, Suklai P, Muralidharan B, Kroll F, Moens TG, Yshii L, Verschoren S, Hölbling BV, Moreira FC, Katona E, Coneys R, de Oliveira P, Zhang YJ, Jansen K, Daughrity LM, McGown A, Ramesh TM, Van Den Bosch L, Lignani G, Rahim AA, Coyne AN, Petrucelli L, Rihel J, Isaacs AM. Kempthorne L, et al. Among authors: roberts mj. Nat Commun. 2025 Jan 8;16(1):459. doi: 10.1038/s41467-024-55550-x. Nat Commun. 2025. PMID: 39779704 Free PMC article.
6,073 results