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, E. D. Belousova, D. V. Vlodavets, L. M. Kuzenkova, S. V. Mikhailova, T. V. Podkletnova, S. G. Popovich, E. V. Uvakina, E. L. Usacheva, С. Б. Артемьева, Е. Д. Белоусова, Д. В. Влодавец, Л. М. Кузенкова, С. В. Михайлова, Т. В. Подклетнова, С. Г. Попович, Е. В. Увакина, Е. Л. Усачева

المساهمون: The study was performed without external funding, Исследование проведено без спонсорской поддержки

المصدر: Medical Genetics; Том 23, № 1 (2024); 19-25 ; Медицинская генетика; Том 23, № 1 (2024); 19-25 ; 2073-7998

مصطلحات موضوعية: антисмысловые олигонуклеотиды, muscular dystrophy, Duchenne muscular dystrophy, exon skipping, casimersen, antisense oligonucleotides, мышечная дистрофия, ПМД Дюшенна, экзон-скиппинг, касимерсен

وصف الملف: application/pdf

العلاقة: https://www.medgen-journal.ru/jour/article/view/2407/1764Test; Drousiotou A., Ioannou P., Georgiou T., et al. Neonatal screening for Duchenne muscular dystrophy: a novel semiquantitative application of the bioluminescence test for creatine kinase in a pilot national program in Cyprus. Genet Test 1998; 2: 55–60.; Emery A.E. Population frequencies of inherited neuromuscular diseases—a world survey. Neuromuscul Disord 1991; 1: 19–29.; Duan D., Goemans N., Takeda S., Mercuri E., Aartsma-Rus A. Duchenne muscular dystrophy. Nat Rev Dis Primers. 2021;7(1):13. doi:10.1038/s41572-021-00248-3.; Happi Mbakam C., Lamothe G., Tremblay J.P. Therapeutic Strategies for Dystrophin Replacement in Duchenne Muscular Dystrophy. Front Med (Lausanne). 2022;9:859930. doi:10.3389/fmed.2022.859930; Hoffman E.P., Brown R.H. Jr, Kunkel L.M. Dystrophin: the protein product of the Duchenne muscular dystrophy locus. Cell 1987; 51: 919–28.; Клинические рекомендации «Прогрессирующая мышечная дистрофия Дюшенна. Прогрессирующая мышечная дистрофия Беккера». https://cr.minzdrav.gov.ru/schema/773Test.; Poysky J., Behavior in DMD Study Group. Behavior patterns in Duchenne muscular dystrophy: report on the Parent Project Muscular Dystrophy behavior workshop 8–9 of December 2006, Philadelphia, USA. Neuromuscul Disord 2007; 17: 986–94.; Ryder S., Leadley R.M., Armstrong N., et al. The burden, epidemiology, costs and treatment for Duchenne muscular dystrophy: an evidence review. Orphanet J Rare Dis. 2017;12(1):79.; Coratti G., Pane M., Brogna C., et al. North Star Ambulatory Assessment changes in ambulant Duchenne boys amenable to skip exons 44, 45, 51, and 53: A 3 year follow up. PLoS One. 2021;16(6):e0253882. doi:10.1371/journal.pone.0253882; de Feraudy Y., Ben Yaou R., Wahbi K., et al.Very Low Residual Dystrophin Quantity Is Associated with Milder Dystrophinopathy. Ann Neurol. 2021;89(2):280-292. doi:10.1002/ana.25951.; Shirley M. Casimersen: First Approval. Drugs. 2021;81(7):875-879. doi:10.1007/s40265-021-01512-2.; Iannaccone S. et al. Casimersen in Patients With Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping: Interim Results From the Phase 3 ESSENCE Trial. World Muscle Society Congress 2022. Neuromuscular Disorders 32 (2022) S42–S136. doi:10.1016/j.nmd.2022.07.248.; Iannaccone S. et al. Casimersen in Patients With Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping: Interim Results From the Phase 3 ESSENCE Trial. Presented at the Muscular Dystrophy Association Clinical & Scientific Conference, March 13–16, 2022, Nashville, TN. https://investorrelations.sarepta.com/static-files/d6ad5b34-752b-4d8b-ba15-bfa88394296fTest Access date 18. 12. 2023; Iannaccone S. et al. Casimersen in Patients With Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping: Interim Results From the Phase 3 ESSENCE Trial. Presented at the 27 th International Hybrid Annual Congress of the World Muscle Society; October 11–15, 2021; Halifax, Nova Scotia, Canada. Neuromuscular Disorders 31 (2021) S47–S162. doi:10.1016/j.nmd.2021.07.175; Iannaccone S. et al. Casimersen in Patients With Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping: Interim Results From the Phase 3 ESSENCE Trial. Presented at the Muscular Dystrophy Association Clinical & Scientific Conference, March 12–22, 2023, Dallas, TX & Virtual. https://www.mdaconference.org/abstract-libraryTest/casimersen-in-patients-with-duchenne-muscular-dystrophy-amenable-to-exon-45-skipping-interim-results-from-the-phase-3-essence-trial/ Access date 18. 12. 2023; Wagner K. et al. Safety, tolerability, and pharmacokinetics of casimersen in patients with Duchenne muscular dystrophy amenable to exon 45 skipping: A randomized, double-blind, placebo-controlled, dose-titration trial. Muscle and Nerve. 2021;64(3):285-292.; NCT02500381. https://clinicaltrials.gov/ct2/show/NCT02500381Test.; NCT02530905. https://clinicaltrials.gov/ct2/show/NCT02530905Test.; NCT03532542. An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy. https://www.clinicaltrials.gov/study/NCT03532542Test.; https://www.medgen-journal.ru/jour/article/view/2407Test

الإتاحة: https://doi.org/10.25557/2073-7998.2024.01.19-25Test
https://doi.org/10.1038/s41572-021-00248-3Test
https://doi.org/10.3389/fmed.2022.859930Test
https://doi.org/10.1371/journal.pone.0253882Test
https://doi.org/10.1002/ana.25951Test
https://doi.org/10.1007/s40265-021-01512-2Test
https://doi.org/10.1016/j.nmd.2022.07.248Test
https://doi.org/10.1016/j.nmd.2021.07.175Test
https://www.medgen-journal.ru/jour/article/view/2407Test

المؤلفون: Milyard Assefa, Addison Gepfert, Meesam Zaheer, Julia M. Hum, Brian W. Skinner

المصدر: Biomedicines, Vol 12, Iss 4, p 912 (2024)

مصطلحات موضوعية: AMONDYS 45, antisense oligonucleotide, casimersen, Duchenne muscular dystrophy, dystrophin, exon skipping, Biology (General), QH301-705.5

وصف الملف: electronic resource

العلاقة: https://www.mdpi.com/2227-9059/12/4/912Test; https://doaj.org/toc/2227-9059Test

الوصول الحر: https://doaj.org/article/f556c363fb6447af92886f221383fa1eTest

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: Vasterling, Megan E, Maitski, Rebecca J, Davis, Brice A, Barnes, Julie E, Kelkar, Rucha A, Klapper, Rachel J, Patel, Hirni, Ahmadzadeh, Shahab, Shekoohi, Sahar, Kaye, Alan D, Varrassi, Giustino

المصدر: School of Medicine Faculty Publications

مصطلحات موضوعية: amondys 45, casimersen, duchenne muscular dystrophy, exon skipping, gene therapy, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Investigative Techniques, Musculoskeletal Diseases, Therapeutics

وصف الملف: application/pdf

العلاقة: https://digitalscholar.lsuhsc.edu/som_facpubs/2129Test; https://digitalscholar.lsuhsc.edu/context/som_facpubs/article/3130/viewcontent/AMONDYS45_Casimersen__2023.pdfTest

الإتاحة: https://doi.org/10.7759/cureus.51237Test
https://digitalscholar.lsuhsc.edu/som_facpubs/2129Test
https://digitalscholar.lsuhsc.edu/context/som_facpubs/article/3130/viewcontent/AMONDYS45_Casimersen__2023.pdfTest

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

المصدر: Pharmaceuticals; Volume 15; Issue 2; Pages: 222

مصطلحات موضوعية: drugs, FDA, oligonucleotides, peptides, antibody-drug conjugate, inclisiran, casimersen, vosoritide, melphalan flufenamide, voclosporin, pegcetacoplan, dasiglucagon, Piflufolastat-F 18, difelikefalin, odevixibat, tisotumab vedotin-tftv, loncastuximab tesirine-lpyl

وصف الملف: application/pdf

العلاقة: Medicinal Chemistry; https://dx.doi.org/10.3390/ph15020222Test

الإتاحة: https://doi.org/10.3390/ph15020222Test

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

مصطلحات موضوعية: FDA, Piflufolastat-F18, antibody-drug conjugate, casimersen, dasiglucagon, difelikefalin, drugs, inclisiran, loncastuximab tesirine-lpyl, melphalan flufenamide, odevixibat, oligonucleotides, pegcetacoplan, peptides, tisotumab vedotin-tftv, voclosporin, vosoritide, 1115 Pharmacology and Pharmaceutical Sciences

العلاقة: Pharmaceuticals; http://hdl.handle.net/10044/1/95772Test

الإتاحة: https://doi.org/10.3390/ph15020222Test
http://hdl.handle.net/10044/1/95772Test

المؤلفون: Matt Shirley (7340599)

مصطلحات موضوعية: Medicine, Neurology and Neuromuscular Diseases, casimersen, Amondys 45, Duchenne Muscular Dystrophy Duchenne, DMD, review, Adis Insight Report

العلاقة: https://figshare.com/articles/online_resource/Casimersen_First_Approval/14312786Test

الإتاحة: https://doi.org/10.6084/m9.figshare.14312786.v1Test

المؤلفون: Fernando Albericio, Danah AlShaer, Beatriz G. De la Torre, Othman Al Musaimi

المصدر: Digital.CSIC. Repositorio Institucional del CSIC
instname

مصطلحات موضوعية: Oligonucleotides, Pharmaceutical Science, vosoritide, Inclisiran, drugs, antibody-drug conjugate, Dasiglucagon, Tisotumab vedotin-tftv, casimersen, Drug Discovery, voclosporin, tisotumab vedotin-tftv, pegcetacoplan, Pegcetacoplan, Antibody-drug conjugate, oligonucleotides, Casimersen, dasiglucagon, Drugs, Difelikefalin, difelikefalin, Voclosporin, Loncastuximab tesirine-lpyl, loncastuximab tesirine-lpyl, Piflufolastat-F18, odevixibat, peptides, Molecular Medicine, 1115 Pharmacology and Pharmaceutical Sciences, melphalan flufenamide, Peptides, inclisiran, FDA, Vosoritide, Melphalan flufenamide

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::2aac2302bffcf06cc3c44760a3cf0203Test
https://pubmed.ncbi.nlm.nih.gov/35215334Test

مصطلحات الفهرس: FDA, Piflufolastat-F18, Antibody-drug conjugate, Casimersen, Dasiglucagon, Difelikefalin, Inclisiran, Vosoritide, Drugs, Voclosporin, Loncastuximab tesirine-lpyl, Melphalan flufenamide, odevixibat, Oligonucleotides, Pegcetacoplan, Peptides, Tisotumab vedotin-tftv, artículo

URL: http://hdl.handle.net/10261/262266Test
https://api.elsevier.com/content/abstract/scopus_id/85124717384Test
https://doi.org/10.3390/ph15020222Test
Pharmaceuticals (Basel, Switzerland)
Publisher's version
https://doi.org/10.3390/ph15020222Test
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