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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: johnson f. 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.
Pharmacokinetic evaluation of single-dose migalastat in non-Fabry disease subjects with ESRD receiving dialysis treatment, and use of modeling to select dose regimens in Fabry disease subjects with ESRD receiving dialysis treatment.
Johnson FK, Wu S, Schmith G, Williams H, Rutecki J, Halabi A, Feldkamp T, Sileno A. Johnson FK, et al. PLoS One. 2024 Dec 5;19(12):e0314030. doi: 10.1371/journal.pone.0314030. eCollection 2024. PLoS One. 2024. PMID: 39636942 Free PMC article.
Patient Preferences for Features Associated With Leadless Versus Conventional Transvenous Cardiac Pacemakers.
Reed SD, Yang JC, Wallace MJ, Sutphin J, Johnson FR, Ozdemir S, Delgado SM, Goates S, Harbert N, Lo M, Rajagopalan B, Ip JE, Al-Khatib SM. Reed SD, et al. Among authors: johnson fr. Circ Cardiovasc Qual Outcomes. 2024 Dec;17(12):e011168. doi: 10.1161/CIRCOUTCOMES.124.011168. Epub 2024 Nov 21. Circ Cardiovasc Qual Outcomes. 2024. PMID: 39569505 Free PMC article.
Disparities in casemix, acute interventions, discharge destinations and mortality of patients with traumatic brain injury between Europe and India.
Gupta D, Singh RD, Vreeburg RJ, van Dijck JT, den Boogert HF, Sharma K, Praneeth K, Clarke DB, Lecky FE, Maas AI, Sinha VD, de Ruiter GC, Peul WC, van Essen TA; CENTER-TBI; CINTER-TBI. Gupta D, et al. J Glob Health. 2024 Nov 11;14:04227. doi: 10.7189/jogh.14.04227. J Glob Health. 2024. PMID: 39526450 Free PMC article.
Cipaglucosidase alfa plus miglustat: linking mechanism of action to clinical outcomes in late-onset Pompe disease.
Byrne BJ, Parenti G, Schoser B, van der Ploeg AT, Do H, Fox B, Goldman M, Johnson FK, Kang J, Mehta N, Mondick J, Sheikh MO, Sitaraman Das S, Tuske S, Brudvig J, Weimer JM, Mozaffar T. Byrne BJ, et al. Among authors: johnson fk. Front Neurol. 2024 Oct 18;15:1451512. doi: 10.3389/fneur.2024.1451512. eCollection 2024. Front Neurol. 2024. PMID: 39494167 Free PMC article.
2,349 results