-
1دورية أكاديمية
المصدر: Frontiers in Neurology, Vol 14 (2023)
مصطلحات موضوعية: Duchene muscular dystrophy (DMD), vamorolone, neuromuscular, glucocorticoids, steroid naïve, Neurology. Diseases of the nervous system, RC346-429
وصف الملف: electronic resource
العلاقة: https://www.frontiersin.org/articles/10.3389/fneur.2023.1107474/fullTest; https://doaj.org/toc/1664-2295Test
-
2دورية أكاديمية
المؤلفون: Susan Keam
مصطلحات موضوعية: Medicine, vamorolone, First Approval, Adis Drug Review, Adis Insight Report, dissociative corticosteroid, Duchenne Muscular Dystrophy
الإتاحة: https://doi.org/10.6084/m9.figshare.24654435.v1Test
https://figshare.com/articles/online_resource/Vamorolone_First_Approval/24654435Test -
3
المؤلفون: Aartsma-Rus, A., Ferlini, A., McNally, E. M., Spitali, P., Sweeney, H. L., Al-Khalili Szigyarto, Cristina, Bello, L., Bronson, A., Brown, K., Buccella, F., Chadwick, J., Frank, D., Hoffman, E., Larkindale, J., McClorey, G., Munschauer, R., Muntoni, F., Owens, J., Schara, U., Straub, V., Tinsley, J., Versnel, J., Vroom, E., Welch, E.
المصدر: Neuromuscular Disorders. 28(1):77-86
مصطلحات موضوعية: Biobank, Biomarker, Duchenne muscular dystrophy, Dystrophin, MRI, alanine aminotransferase, aspartate aminotransferase, biological marker, carbonate dehydratase III, corticosteroid, dysferlin, enolase, fukutin related protein, glucocorticoid receptor, glutamate dehydrogenase, immunoglobulin enhancer binding protein, microRNA, microRNA 133a, microRNA 133b, osteopontin, protein enolase 3, stromelysin, unclassified drug, utrophin, vamorolone, alanine aminotransferase level, aspartate aminotransferase blood level, Becker muscular dystrophy, blood analysis, blood sampling, Conference Paper, corticosteroid therapy, CRISPR-CAS9 system, DNA modification, drug design, exon, exon skipping, gene, gene expression, human, lipidomics, LTBP4 gene, Netherlands, nonhuman, nuclear magnetic resonance imaging, priority journal, protein expression, real time polymerase chain reaction, single nucleotide polymorphism, SPP1 gene, urinalysis, urine sampling, workshop, animal, information dissemination, male, metabolism, non profit organization, validation study, Animals, Biomarkers, Humans, Muscular Dystrophy, Duchenne, Organizations, Nonprofit, Validation Studies as Topic
وصف الملف: print
الوصول الحر: https://urn.kb.se/resolve?urn=urn:nbn:se:kth:diva-282709Test
https://doi.org/10.1016/j.nmd.2017.10.002Test -
4دورية أكاديمية
المؤلفون: Mukesh Kumar, Venugopalan Y Vishnu
المصدر: Journal of Current Research in Scientific Medicine, Vol 5, Iss 2, Pp 78-84 (2019)
مصطلحات موضوعية: duchenne muscular dystrophy, exon skipping, nusinersen, recent advances, risdiplam, spinal muscular atrophy, vamorolone, zolgensma, Medicine
وصف الملف: electronic resource
العلاقة: http://www.jcrsmed.org/article.asp?issn=2455-3069;year=2019;volume=5;issue=2;spage=78;epage=84;aulast=KumarTest; https://doaj.org/toc/2455-3069Test
-
5تقرير
المؤلفون: Dr Laurie Conklin, Dr Holly Peay, Suzanne Gaglianone, Suzie-Ann Bakker, Elizabeth Vroom, Dr Eric Hoffman, Christina Olsen
مصطلحات موضوعية: Duchenne muscular dystrophy, clinical trial, VISION-DMD, return of patient data, vamorolone
العلاقة: info:eu-repo/grantAgreement/EC/H2020/667078/; https://zenodo.org/communities/vision-dmd-h2020Test; https://zenodo.org/record/4836583Test; https://doi.org/10.5281/zenodo.4836583Test; oai:zenodo.org:4836583
الإتاحة: https://doi.org/10.5281/zenodo.4836583Test
https://doi.org/10.5281/zenodo.4836582Test
https://zenodo.org/record/4836583Test -
6تقرير
المؤلفون: Christina Olsen, Ritchie Head, Jane Knerova, Jane Larkindale, Susan Ward, Pietro Spitali, Hermien Kan
مصطلحات موضوعية: Duchenne muscular dystrophy, Vamorolone, Biomarkers
العلاقة: info:eu-repo/grantAgreement/EC/H2020/667078/; https://zenodo.org/communities/vision-dmd-h2020Test; https://zenodo.org/record/5668693Test; https://doi.org/10.5281/zenodo.5668693Test; oai:zenodo.org:5668693
الإتاحة: https://doi.org/10.5281/zenodo.5668693Test
https://doi.org/10.5281/zenodo.5668575Test
https://zenodo.org/record/5668693Test -
7دورية أكاديمية
المؤلفون: Hoffman, Eric P., Schwartz, Benjamin D., Mengle-Gaw, Laurel J., Smith, Edward C., Castro, Diana, Mah, Jean K., McDonald, Craig M., Kuntz, Nancy L., Finkel, Richard S., Guglieri, Michela, Bushby, Katharine, Tulinius, Mar, Nevo, Yoram, Ryan, Monique M., Webster, Richard, Smith, Andrea L., Morgenroth, Lauren P., Arrieta, Adrienne, Shimony, Maya, Siener, Catherine, Jaros, Mark, Shale, Phil, McCall, John M., Nagaraju, Kanneboyina, van den Anker, John, Conklin, Laurie S., Cnaan, Avital, Gordish-Dressman, Heather, Damsker, Jesse M., Clemens, Paula R.
مصطلحات موضوعية: Clinical trials Observational study (Cohort, Case control), Class IV, Muscle disease, Duchenne muscular dystrophy, vamorolone
جغرافية الموضوع: United States, United Kingdom, Sweden, Israel, Australia
الوقت: Canada
العلاقة: Hoffman EP, Schwartz BD, Mengle-Gaw LJ, Smith EC, Castro D, Mah JK, McDonald CM, Kuntz NL, Finkel RS, Guglieri M, Bushby K, Tulinius M, Nevo Y, Ryan MM, Webster R, Smith AL, Morgenroth LP, Arrieta A, Shimony M, Siener C, Jaros M, Shale P, McCall JM, Nagaraju K, van den Anker J, Conklin LS, Cnaan A, Gordish-Dressman H, Damsker JM, Clemens PR (2019) Vamorolone trial in Duchenne muscular dystrophy shows dose-related improvement of muscle function. Neurology.; http://hdl.handle.net/10255/dryad.208749Test
الإتاحة: https://doi.org/10.5061/dryad.1rd4hc7Test
https://doi.org/10.5061/dryad.1rd4hc7/2Test
https://doi.org/10.1212/WNL.0000000000008168Test
http://hdl.handle.net/10255/dryad.208749Test -
8مورد إلكتروني
مصطلحات الفهرس: Biobank, Biomarker, Duchenne muscular dystrophy, Dystrophin, MRI, alanine aminotransferase, aspartate aminotransferase, biological marker, carbonate dehydratase III, corticosteroid, dysferlin, enolase, fukutin related protein, glucocorticoid receptor, glutamate dehydrogenase, immunoglobulin enhancer binding protein, microRNA, microRNA 133a, microRNA 133b, osteopontin, protein enolase 3, stromelysin, unclassified drug, utrophin, vamorolone, alanine aminotransferase level, aspartate aminotransferase blood level, Becker muscular dystrophy, blood analysis, blood sampling, Conference Paper, corticosteroid therapy, CRISPR-CAS9 system, DNA modification, drug design, exon, exon skipping, gene, gene expression, human, lipidomics, LTBP4 gene, Netherlands, nonhuman, nuclear magnetic resonance imaging, priority journal, protein expression, real time polymerase chain reaction, single nucleotide polymorphism, SPP1 gene, urinalysis, urine sampling, workshop, animal, information dissemination, male, metabolism, non profit organization, validation study, Animals, Biomarkers, Humans, Muscular Dystrophy, Duchenne, Organizations, Nonprofit, Validation Studies as Topic, Cell and Molecular Biology, Cell- och molekylärbiologi, Article in journal, info:eu-repo/semantics/article, text
URL:
http://urn.kb.se/resolve?urn=urn:nbn:se:kth:diva-282709Test
Neuromuscular Disorders, 0960-8966, 2018, 28:1, s. 77-86