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989 results

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Page 1
Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial.
Mercuri E, Vilchez JJ, Boespflug-Tanguy O, Zaidman CM, Mah JK, Goemans N, Müller-Felber W, Niks EH, Schara-Schmidt U, Bertini E, Comi GP, Mathews KD, Servais L, Vandenborne K, Johannsen J, Messina S, Spinty S, McAdam L, Selby K, Byrne B, Laverty CG, Carroll K, Zardi G, Cazzaniga S, Coceani N, Bettica P, McDonald CM; EPIDYS Study Group. Mercuri E, et al. Among authors: byrne b. Lancet Neurol. 2024 Apr;23(4):393-403. doi: 10.1016/S1474-4422(24)00036-X. Lancet Neurol. 2024. PMID: 38508835 Clinical Trial.
Eteplirsen for the treatment of Duchenne muscular dystrophy.
Mendell JR, Rodino-Klapac LR, Sahenk Z, Roush K, Bird L, Lowes LP, Alfano L, Gomez AM, Lewis S, Kota J, Malik V, Shontz K, Walker CM, Flanigan KM, Corridore M, Kean JR, Allen HD, Shilling C, Melia KR, Sazani P, Saoud JB, Kaye EM; Eteplirsen Study Group. Mendell JR, et al. Ann Neurol. 2013 Nov;74(5):637-47. doi: 10.1002/ana.23982. Epub 2013 Sep 10. Ann Neurol. 2013. PMID: 23907995 Free article. Clinical Trial.
Towards regulatory endorsement of drug development tools to promote the application of model-informed drug development in Duchenne muscular dystrophy.
Conrado DJ, Larkindale J, Berg A, Hill M, Burton J, Abrams KR, Abresch RT, Bronson A, Chapman D, Crowther M, Duong T, Gordish-Dressman H, Harnisch L, Henricson E, Kim S, McDonald CM, Schmidt S, Vong C, Wang X, Wong BL, Yong F, Romero K; Duchenne Muscular Dystrophy Regulatory Science Consortium (D-RSC). Conrado DJ, et al. J Pharmacokinet Pharmacodyn. 2019 Oct;46(5):441-455. doi: 10.1007/s10928-019-09642-7. Epub 2019 May 24. J Pharmacokinet Pharmacodyn. 2019. PMID: 31127458
The Clinical Development of Taldefgrobep Alfa: An Anti-Myostatin Adnectin for the Treatment of Duchenne Muscular Dystrophy.
Muntoni F, Byrne BJ, McMillan HJ, Ryan MM, Wong BL, Dukart J, Bansal A, Cosson V, Dreghici R, Guridi M, Rabbia M, Staunton H, Tirucherai GS, Yen K, Yuan X, Wagner KR; Taldefgrobep Alfa Study Group. Muntoni F, et al. Neurol Ther. 2024 Feb;13(1):183-219. doi: 10.1007/s40120-023-00570-w. Epub 2024 Jan 8. Neurol Ther. 2024. PMID: 38190001 Free PMC article.
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.
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.
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.
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
The dimer of human SVCT1 is key for transport function.
Woubshete M, Chan LI, Diallinas G, Byrne B. Woubshete M, et al. Among authors: byrne b. Biochim Biophys Acta Biomembr. 2024 Dec;1866(8):184390. doi: 10.1016/j.bbamem.2024.184390. Epub 2024 Oct 4. Biochim Biophys Acta Biomembr. 2024. PMID: 39369805 Free article.
989 results