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Page 1
Urinary titin reflects the severity of walking ability, muscle strength, and muscle and cardiac damage in patients with Becker muscular dystrophy.
Awano H, Nambu Y, Osawa K, Shirakawa T, Matsumura T, Wakisaka A, Kuru S, Funato M, Takeshima Y, Ishigaki K, Kobayashi M, Sato T, Fujii T, Sugie K, Kimura K, Komaki H, Nakamura A, Matsuo M. Awano H, et al. Among authors: komaki h. Clin Chim Acta. 2025 Jan 30;566:120053. doi: 10.1016/j.cca.2024.120053. Epub 2024 Nov 17. Clin Chim Acta. 2025. PMID: 39561886
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.
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
Clinical Characteristics of Patients With Becker Muscular Dystrophy Having Pathogenic Microvariants or Duplications.
Nakamura A, Matsumura T, Ogata K, Mori-Yoshimura M, Takeshita E, Kimura K, Arahata H, Takeshima Y, Takahashi T, Ishigaki K, Awano H, Sugie K, Fujii T, Oi H, Komaki H. Nakamura A, et al. Among authors: komaki h. Neurol Genet. 2024 Dec 17;11(1):e200215. doi: 10.1212/NXG.0000000000200215. eCollection 2025 Feb. Neurol Genet. 2024. PMID: 39712290 Free PMC article.
Elevated plasma progranulin levels in the acute phase are correlated with recovery of left ventricular function in the chronic phase in patients with acute myocardial infarction.
Minatoguchi S, Satake A, Murase H, Yoshizumi R, Komaki H, Baba S, Yasuda S, Ojio S, Tanaka T, Okura H, Minatoguchi S. Minatoguchi S, et al. Among authors: komaki h. PLoS One. 2024 Nov 8;19(11):e0313014. doi: 10.1371/journal.pone.0313014. eCollection 2024. PLoS One. 2024. PMID: 39514465 Free PMC article.
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. 2024 Oct 9. doi: 10.1038/s41591-024-03304-z. Online ahead of print. Nat Med. 2024. PMID: 39385046
375 results