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288 results

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104-week efficacy and safety of cipaglucosidase alfa plus miglustat in adults with late-onset Pompe disease: a phase III open-label extension study (ATB200-07).
Schoser B, Kishnani PS, Bratkovic D, Byrne BJ, Claeys KG, Díaz-Manera J, Laforêt P, Roberts M, Toscano A, van der Ploeg AT, Castelli J, Goldman M, Holdbrook F, Sitaraman Das S, Wasfi Y, Mozaffar T; ATB200-07 Study Group. Schoser B, et al. Among authors: byrne bj. J Neurol. 2024 May;271(5):2810-2823. doi: 10.1007/s00415-024-12236-0. Epub 2024 Feb 28. J Neurol. 2024. PMID: 38418563 Free PMC article. Clinical Trial.
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. Front Neurol. 2024 Oct 18;15:1451512. doi: 10.3389/fneur.2024.1451512. eCollection 2024. Front Neurol. 2024. PMID: 39494167 Free PMC article.
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: byrne bj. 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.
Differences in Pediatricians' Income by Sex Over Time.
Frintner MP, Freed GL, Byrne BJ, Leslie LK, Starmer AJ, Gottschlich EA, Olson LM. Frintner MP, et al. Among authors: byrne bj. Pediatrics. 2024 Nov 1;154(5):e2024066240. doi: 10.1542/peds.2024-066240. Pediatrics. 2024. PMID: 39468944
Taldefgrobep Alfa and the Phase 3 RESILIENT Trial in Spinal Muscular Atrophy.
Servais L, Lair LL, Connolly AM, Byrne BJ, Chen KS, Coric V, Qureshi I, Durham S, Campbell DJ, Maclaine G, Marin J, Bechtold C. Servais L, et al. Among authors: byrne bj. Int J Mol Sci. 2024 Sep 24;25(19):10273. doi: 10.3390/ijms251910273. Int J Mol Sci. 2024. PMID: 39408601 Free PMC article. Review.
Neurological glycogen storage diseases and emerging therapeutics.
Colpaert M, Singh PK, Donohue KJ, Pires NT, Fuller DD, Corti M, Byrne BJ, Sun RC, Vander Kooi CW, Gentry MS. Colpaert M, et al. Among authors: byrne bj. Neurotherapeutics. 2024 Sep;21(5):e00446. doi: 10.1016/j.neurot.2024.e00446. Epub 2024 Sep 14. Neurotherapeutics. 2024. PMID: 39277505 Free PMC article. Review.
Codon-Optimized and de novo-Synthesized E-Selectin/AAV2 Dose-Response Study for Vascular Regeneration Gene Therapy.
Voza FA, Byrne BJ, Ortiz YY, Li Y, Le N, Osafo L, Ribieras AC, Shao H, Huerta CT, Wei Y, Falero-Diaz G, Franco-Bravo A, Lassance-Soares RM, Vazquez-Padron RI, Liu ZJ, Velazquez OC. Voza FA, et al. Among authors: byrne bj. Ann Surg. 2024 Oct 1;280(4):570-583. doi: 10.1097/SLA.0000000000006436. Epub 2024 Jul 8. Ann Surg. 2024. PMID: 38975668 Free PMC article.
Innate Immune Sensing of Adeno-Associated Virus Vectors.
Cao D, Byrne BJ, de Jong YP, Terhorst C, Duan D, Herzog RW, Kumar SRP. Cao D, et al. Among authors: byrne bj. Hum Gene Ther. 2024 Jul;35(13-14):451-463. doi: 10.1089/hum.2024.040. Epub 2024 Jul 5. Hum Gene Ther. 2024. PMID: 38887999 Review.
288 results