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Page 1
AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trial.
Mendell JR, Muntoni F, McDonald CM, Mercuri EM, Ciafaloni E, Komaki H, Leon-Astudillo C, Nascimento A, Proud C, Schara-Schmidt U, Veerapandiyan A, Zaidman CM, Guridi M, Murphy AP, Reid C, Wandel C, Asher DR, Darton E, Mason S, Potter RA, Singh T, Zhang W, Fontoura P, Elkins JS, Rodino-Klapac LR. Mendell JR, et al. Among authors: komaki h. Nat Med. 2025 Jan;31(1):332-341. doi: 10.1038/s41591-024-03304-z. Epub 2024 Oct 9. Nat Med. 2025. PMID: 39385046 Free PMC article. Clinical Trial.
A phase 3 randomized placebo-controlled trial of tadalafil for Duchenne muscular dystrophy.
Victor RG, Sweeney HL, Finkel R, McDonald CM, Byrne B, Eagle M, Goemans N, Vandenborne K, Dubrovsky AL, Topaloglu H, Miceli MC, Furlong P, Landry J, Elashoff R, Cox D; Tadalafil DMD Study Group. Victor RG, et al. Neurology. 2017 Oct 24;89(17):1811-1820. doi: 10.1212/WNL.0000000000004570. Epub 2017 Sep 29. Neurology. 2017. PMID: 28972192 Free PMC article. Clinical Trial.
A randomized placebo-controlled phase 3 trial of an antisense oligonucleotide, drisapersen, in Duchenne muscular dystrophy.
Goemans N, Mercuri E, Belousova E, Komaki H, Dubrovsky A, McDonald CM, Kraus JE, Lourbakos A, Lin Z, Campion G, Wang SX, Campbell C; DEMAND III study group. Goemans N, et al. Among authors: komaki h. Neuromuscul Disord. 2018 Jan;28(1):4-15. doi: 10.1016/j.nmd.2017.10.004. Epub 2017 Dec 6. Neuromuscul Disord. 2018. PMID: 29203355 Free article. Clinical Trial.
A phase I study of TAS-205 in patients with Duchenne muscular dystrophy.
Takeshita E, Komaki H, Shimizu-Motohashi Y, Ishiyama A, Sasaki M, Takeda S. Takeshita E, et al. Among authors: komaki h. Ann Clin Transl Neurol. 2018 Oct 10;5(11):1338-1349. doi: 10.1002/acn3.651. eCollection 2018 Nov. Ann Clin Transl Neurol. 2018. PMID: 30480028 Free PMC article.
International retrospective natural history study of LMNA-related congenital muscular dystrophy.
Ben Yaou R, Yun P, Dabaj I, Norato G, Donkervoort S, Xiong H, Nascimento A, Maggi L, Sarkozy A, Monges S, Bertoli M, Komaki H, Mayer M, Mercuri E, Zanoteli E, Castiglioni C, Marini-Bettolo C, D'Amico A, Deconinck N, Desguerre I, Erazo-Torricelli R, Gurgel-Giannetti J, Ishiyama A, Kleinsteuber KS, Lagrue E, Laugel V, Mercier S, Messina S, Politano L, Ryan MM, Sabouraud P, Schara U, Siciliano G, Vercelli L, Voit T, Yoon G, Alvarez R, Muntoni F, Pierson TM, Gómez-Andrés D, Reghan Foley A, Quijano-Roy S, Bönnemann CG, Bonne G. Ben Yaou R, et al. Among authors: komaki h. Brain Commun. 2021 Apr 11;3(3):fcab075. doi: 10.1093/braincomms/fcab075. eCollection 2021 Jul. Brain Commun. 2021. PMID: 34240052 Free PMC article.
Safety and efficacy of once-daily risdiplam in type 2 and non-ambulant type 3 spinal muscular atrophy (SUNFISH part 2): a phase 3, double-blind, randomised, placebo-controlled trial.
Mercuri E, Deconinck N, Mazzone ES, Nascimento A, Oskoui M, Saito K, Vuillerot C, Baranello G, Boespflug-Tanguy O, Goemans N, Kirschner J, Kostera-Pruszczyk A, Servais L, Gerber M, Gorni K, Khwaja O, Kletzl H, Scalco RS, Staunton H, Yeung WY, Martin C, Fontoura P, Day JW; SUNFISH Study Group. Mercuri E, et al. Lancet Neurol. 2022 Jan;21(1):42-52. doi: 10.1016/S1474-4422(21)00367-7. Lancet Neurol. 2022. PMID: 34942136
Decreased resting energy expenditure in patients with Duchenne muscular dystrophy.
Shimizu-Fujiwara M, Komaki H, Nakagawa E, Mori-Yoshimura M, Oya Y, Fujisaki T, Tokita Y, Kubota N, Shimazaki R, Sato K, Ishikawa T, Goto K, Mochizuki H, Takanoha S, Ogata K, Kawai M, Konagaya M, Miyazaki T, Tatara K, Sugai K, Sasaki M. Shimizu-Fujiwara M, et al. Among authors: komaki h. Brain Dev. 2012 Mar;34(3):206-12. doi: 10.1016/j.braindev.2011.05.005. Epub 2011 May 31. Brain Dev. 2012. PMID: 21632191
Efficacy and tolerance of gastrostomy feeding in Japanese muscular dystrophy patients.
Mizuno T, Komaki H, Sasaki M, Takanoha S, Kuroda K, Kon K, Mamiya S, Yoshioka M, Yatabe K, Mikata T, Ishihara T, Nakajima T, Watanabe H, Konagaya M, Mitani M, Konishi T, Tokita Y, Fukuda K, Tatara K, Maruta K, Imamura S, Shimazaki R, Ishikawa K, Saito T, Shinno S. Mizuno T, et al. Among authors: komaki h. Brain Dev. 2012 Oct;34(9):756-62. doi: 10.1016/j.braindev.2011.11.012. Epub 2011 Dec 26. Brain Dev. 2012. PMID: 22204944
Prednisolone improves walking in Japanese Duchenne muscular dystrophy patients.
Takeuchi F, Yonemoto N, Nakamura H, Shimizu R, Komaki H, Mori-Yoshimura M, Hayashi YK, Nishino I, Kawai M, Kimura E, Takeda S. Takeuchi F, et al. Among authors: komaki h. J Neurol. 2013 Dec;260(12):3023-9. doi: 10.1007/s00415-013-7104-y. Epub 2013 Sep 22. J Neurol. 2013. PMID: 24057148 Free PMC article.
379 results