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Sildenafil does not improve cardiomyopathy in Duchenne/Becker muscular dystrophy.
Leung DG, Herzka DA, Thompson WR, He B, Bibat G, Tennekoon G, Russell SD, Schuleri KH, Lardo AC, Kass DA, Thompson RE, Judge DP, Wagner KR. Leung DG, et al. Among authors: bibat g. Ann Neurol. 2014 Oct;76(4):541-9. doi: 10.1002/ana.24214. Epub 2014 Jul 10. Ann Neurol. 2014. PMID: 25042693 Free PMC article. Clinical Trial.
Randomized open-label trial of dextromethorphan in Rett syndrome.
Smith-Hicks CL, Gupta S, Ewen JB, Hong M, Kratz L, Kelley R, Tierney E, Vaurio R, Bibat G, Sanyal A, Yenokyan G, Brereton N, Johnston MV, Naidu S. Smith-Hicks CL, et al. Among authors: bibat g. Neurology. 2017 Oct 17;89(16):1684-1690. doi: 10.1212/WNL.0000000000004515. Epub 2017 Sep 20. Neurology. 2017. PMID: 28931647 Free PMC article. Clinical Trial.
The NIH Toolbox for cognitive surveillance in Duchenne muscular dystrophy.
Thangarajh M, Kaat AJ, Bibat G, Mansour J, Summerton K, Gioia A, Berger C, Hardy KK, Wagner KR. Thangarajh M, et al. Among authors: bibat g. Ann Clin Transl Neurol. 2019 Sep;6(9):1696-1706. doi: 10.1002/acn3.50867. Epub 2019 Aug 31. Ann Clin Transl Neurol. 2019. PMID: 31472009 Free PMC article.
Clinical trial readiness to solve barriers to drug development in FSHD (ReSolve): protocol of a large, international, multi-center prospective study.
LoRusso S, Johnson NE, McDermott MP, Eichinger K, Butterfield RJ, Carraro E, Higgs K, Lewis L, Mul K, Sacconi S, Sansone VA, Shieh P, van Engelen B, Wagner K, Wang L, Statland JM, Tawil R; ReSolve Investigators and the FSHD CTRN18. LoRusso S, et al. BMC Neurol. 2019 Sep 10;19(1):224. doi: 10.1186/s12883-019-1452-x. BMC Neurol. 2019. PMID: 31506080 Free PMC article.
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.
32 results