المؤلفون: Smith, Edward, Zaidman, Craig, Nakagawa, Tomoyuki, Hoffman, Eric, Clemens, Paula, Rao, Vamshi, Connolly, Anne, Harper, Amy, Mah, Jean, McDonald, Craig

المصدر: Journal of Neuromuscular Diseases. 10(3)

مصطلحات موضوعية: Duchenne muscular dystrophy, clinical efficacy, dystrophin, exon skipping, viltolarsen, Male, Humans, Muscular Dystrophy, Duchenne, Dystrophin, Oligonucleotides, Glucocorticoids

وصف الملف: application/pdf

الوصول الحر: https://escholarship.org/uc/item/57f918sqTest

المؤلفون: Umme Sabrina Haque, Melissa Kohut, Toshifumi Yokota

المصدر: Current Research in Toxicology, Vol 7, Iss , Pp 100182- (2024)

مصطلحات موضوعية: DMD, Eteplirsen, Golodirsen, Viltolarsen, Casimersen, SRP-5051, Toxicology. Poisons, RA1190-1270

وصف الملف: electronic resource

العلاقة: http://www.sciencedirect.com/science/article/pii/S2666027X24000355Test; https://doaj.org/toc/2666-027XTest

الوصول الحر: https://doaj.org/article/4bc8b59de5f7483da8fd0386aaf8f26cTest

المؤلفون: S. B. Artemyeva, О. А. Shidlovskaya, Yu. О. Papina, А. V. Monakhova, I. V. Shulyakov, D. V. Vlodavets, С. Б. Артемьева, О. А. Шидловская, Ю. О. Папина, А. В. Монахова, И. В. Шулякова, Д. В. Влодавец

المصدر: Neuromuscular Diseases; Том 13, № 4 (2023); 103‑112 ; Нервно-мышечные болезни; Том 13, № 4 (2023); 103‑112 ; 2413-0443 ; 2222-8721 ; 10.17650/2222-8721-2023-13-4

مصطلحات موضوعية: вилтоларсен, DMD, dystrophin, exon-skipping, antisense nucleotides, viltolarsen, дистрофин, экзон-скиппинг, антисмысловые нуклеотиды

وصف الملف: application/pdf

العلاقة: https://nmb.abvpress.ru/jour/article/view/578/371Test; Клинические рекомендации «Прогрессирующая мышечная дистрофия Дюшенна. Прогрессирующая мышечная дистрофия Беккера». 2023.; Гремяковa Т.А. Диагностика миодистрофии Дюшенна: проблемы и решения со стороны пациентского сообщества. Доступно по: http://www.dmd-russia.ru/konferentsii/conference/conference-session-1Test/.; Gao Q., McNelly E.M. The dystrophin complex: structure, function and implications for therapy. Compr Physiol 2015;5(3):1223–39. DOI:10.1002/cphy.c140048; Blake D.J., Weir A., Newey S.E. et al. Function and genetics of dystrophin and dystrophin-related proteins in muscle. Physiol Rev 2002;82(2):291–329. DOI:10.1152/physrev.00028.2001; Werneck L.C., Lorenzoni P.J., Dal-Prá Ducci R. et al. Duchenne muscular dystrophy: An historical treatment review. Arq Neuropsiquiatr 2019;77(8):579–89. DOI:10.1590/0004-282X20190088; Frank D.E., et al. Increased dystrophin production with golodirsen in patients with Duchenne muscular dystrophy. Neurology 2020;94(21):e2270–e2282.; Mercuri E., Seferian A.M., Servais L. et al. Safety, tolerability and pharmacokinetics of eteplirsen in young boys aged 6–48 months with Duchenne muscular dystrophy amenable to exon 51 skipping. Neuromuscul Disord 2023;33(6):476–83. DOI:10.1016/j.nmd.2023.03.008.; Clemens P.R., Rao V.K., Connollyet A.M. et al. Safety, tolerability, and efficacy of viltolarsen in boys with Duchenne muscular dystrophy amenable to exon 53 skipping. A phase 2 randomized clinical trial. JAMA Neurol 2020;77(8):982–91. DOI:10.1001/jamaneurol.2020.1264; Iannaoccone S., Phan H., Straub V. et al. Casimersen in patients with Duchenne muscular dystrophy amenable to exon 45 skipping: Interim results from the phase 3 ESSENCE trial. Neuromuscul Disord 2022;32(1).; Mercuri E., Muntoni F., Nascimento Osorio A. et al. Safety and effectiveness of ataluren: Comparison of results from the STRIDE Registry and CINRG DMD Natural History Study. J Comp Eff Res 2020;9(5):341–60. DOI:10.2217/cer-2019-0171.; Komaki H., Nagata T., Saito T. et al. Systemic administration of the antisense oligonucleotide NS-065/NCNP-01 for skipping of exon 53 in patients with Duchenne muscular dystrophy. Sci Transl Med 2018;10(437):eaan0713. DOI:10.1126/scitranslmed.aan0713; Clemens P.R., Rao V.K., Connolly A.M. et al. Efficacy and safety of viltolarsen in boys with Duchenne muscular dystrophy: results from the phase 2, open-label, 4-year extension study. J Neuromuscul Dis 2023;10(3):439–47. DOI:10.3233/JND-221656; https://nmb.abvpress.ru/jour/article/view/578Test

الإتاحة: https://doi.org/10.17650/2222-8721-2023-13-4-103-112Test
https://doi.org/10.17650/2222-8721-2023-13-4Test
https://doi.org/10.1002/cphy.c140048Test
https://doi.org/10.1152/physrev.00028.2001Test
https://doi.org/10.1590/0004-282X20190088Test
https://doi.org/10.1016/j.nmd.2023.03.008Test
https://doi.org/10.1001/jamaneurol.2020.1264Test
https://doi.org/10.2217/cer-2019-0171Test
https://doi.org/10.1126/scitranslmed.aan0713Test
https://doi.org/10.3233/JND-221656Test

المؤلفون: Clemens, Paula, Rao, Vamshi, Connolly, Anne, Harper, Amy, Mah, Jean, Smith, Edward, Zaidman, Craig, Nakagawa, Tomoyuki, Hoffman, Eric, McDonald, Craig

المصدر: Journal of Neuromuscular Diseases. 9(4)

مصطلحات موضوعية: Duchenne muscular dystrophy, clinical efficacy, dystrophin, exon skipping, viltepso, viltolarsen, Dystrophin, Humans, Male, Muscular Dystrophy, Duchenne, Oligonucleotides, Oligonucleotides, Antisense

وصف الملف: application/pdf

الوصول الحر: https://escholarship.org/uc/item/8165p6twTest

المؤلفون: Harry Wilton-Clark, Toshifumi Yokota

المصدر: Pharmaceutics; Volume 15; Issue 3; Pages: 778

مصطلحات موضوعية: eteplirsen, golodirsen, viltolarsen, casimersen, WVE-N531, SRP-5051, DS-5141B, NS-089/NCNP-02, SCAAV9.U7.ACCA, ATL1102

وصف الملف: application/pdf

العلاقة: Gene and Cell Therapy; https://dx.doi.org/10.3390/pharmaceutics15030778Test

الإتاحة: https://doi.org/10.3390/pharmaceutics15030778Test

المؤلفون: Clemens, Paula R, Rao, Vamishi K, Connolly, Anne M, Harper, Amy D, Mah, Jean K, McDonald, Craig M, Smith, Edward C, Zaidman, Craig M, Nakagawa, Tomoyuki, the CINRG DNHS Investigators, Hoffman, Eric P

المصدر: Pharmacy Faculty Scholarship

مصطلحات موضوعية: Duchenne muscular dystrophy, dystrophin, viltolarsen, exon skipping, clinical efficacy

وصف الملف: application/pdf

العلاقة: https://orb.binghamton.edu/pharmacy_fac/4Test; https://orb.binghamton.edu/context/pharmacy_fac/article/1003/viewcontent/jnd_2023_10_3_jnd_10_3_jnd221656_jnd_10_jnd221656.pdfTest

الإتاحة: https://orb.binghamton.edu/pharmacy_fac/4Test
https://orb.binghamton.edu/context/pharmacy_fac/article/1003/viewcontent/jnd_2023_10_3_jnd_10_3_jnd221656_jnd_10_jnd221656.pdfTest

المؤلفون: Kotaro Yoshioka, Maho Katsuyama, Takanori Yokota, 勝山 真帆, 吉岡 耕太郎, 横田 隆徳

المصدر: 神経治療学 / Neurological Therapeutics. 2023, 40(3):237

المؤلفون: Norio Motohashi, Yoshitsugu Aoki, 本橋 紀夫, 青木 吉嗣

المصدر: 神経治療学 / Neurological Therapeutics. 2022, 39(4):430

المؤلفون: Othman Al Musaimi, Danah Al Shaer, Fernando Albericio, Beatriz de la Torre

المصدر: Pharmaceuticals; Volume 14; Issue 2; Pages: 145

مصطلحات موضوعية: belantamab mafodotin-blmf, 64 Cu-DOTATATE, drugs, FDA, 68 Ga-PSMA-11, lumasiran, setmelanotide, oligonucleotides, peptides, viltolarsen

وصف الملف: application/pdf

العلاقة: Medicinal Chemistry; https://dx.doi.org/10.3390/ph14020145Test

الإتاحة: https://doi.org/10.3390/ph14020145Test

المؤلفون: Al Musaimi, O, Al Shaer, D, Albericio, F, De la Torre, BG

مصطلحات موضوعية: Science & Technology, Life Sciences & Biomedicine, Chemistry, Medicinal, Pharmacology & Pharmacy, belantamab mafodotin-blmf, Cu-64-DOTATATE, drugs, FDA, Ga-68-PSMA-11, lumasiran, setmelanotide, oligonucleotides, peptides, viltolarsen, 64Cu-DOTATATE, 68Ga-PSMA-11, 1115 Pharmacology and Pharmaceutical Sciences

العلاقة: Pharmaceuticals; http://hdl.handle.net/10044/1/94838Test

الإتاحة: https://doi.org/10.3390/ph14020145Test
http://hdl.handle.net/10044/1/94838Test