المؤلفون: Rowan H. Elhalag, Karam R. Motawea, Nesreen Elsayed Talat, Samah S. Rouzan, Jaffer Shah

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

الوصف: Background and aimRecent studies evaluated the role of vamorolone in treating Duchenne muscular dystrophy (DMD), so we aimed in our Meta-analysis to assess the efficacy of vamorolone in comparison with placebo and corticosteroids for treating DMD patients.MethodsWe searched PubMed, Web of Science, Scopus, and Cochrane library databases. We included any randomized control trials and controlled observational studies that investigated the role of vamorolone in treating DMD patients. We used RevMan software, version 5.4. to perform our meta-analysis.ResultsAfter a search of the literature, 4 studies were included in the meta-analysis; the total number of patients included in the study is 277 patients, 125 patients in the vamorolone group, 106 in the glucocorticoids group, and 46 in placebo (steroid naïve) group. The pooled analysis showed a statistically significant association between the vamorolone group and increased TTSTAND velocity, TTRW velocity and TTCLIMB velocity compared with the placebo group (MD = 0.04, 95% CI = 0.02–0.07, p = 0.002), (MD = 0.24, 95% CI = 0.11–0.37, p = 0.0003), and (MD = 0.06, 95% CI = 0.05–0.06, p < 0.00001), respectively. Also, the analysis showed a statistically significant association between vamorolone and increased TTRW velocity and increased Height percentile for age compared with the glucocorticoid group (MD = −0.14, 95% CI = −0.26 to −0.01, p = 0.03) and (MD = 17.82, 95% CI = 3.89–31.75, p = 0.01), respectively.ConclusionOur study revealed a significant association between vamorolone and increased TTSTAND velocity, TTRW velocity, and TTCLIMB velocity compared with the placebo (steroid naïve), also showed a statistically significant association between increased TTRW velocity and increased Height percentile for age compared with the glucocorticoid that enhances the privilege of vamorolone over glucocorticoid in treating DMD patients. More multicenter randomized studies are needed to support our results.

وصف الملف: electronic resource

العلاقة: https://www.frontiersin.org/articles/10.3389/fneur.2023.1107474/fullTest; https://doaj.org/toc/1664-2295Test

الوصول الحر: https://doaj.org/article/02329c1ecd454f298524b33521db0c1bTest

المؤلفون: Susan Keam

مصطلحات موضوعية: Medicine, vamorolone, First Approval, Adis Drug Review, Adis Insight Report, dissociative corticosteroid, Duchenne Muscular Dystrophy

الوصف: Declarations Funding The preparation of this review was not supported by any external funding. Authorship and Conflict of interest During the peer review process the manufacturer of the agent under review was offered an opportunity to comment on the article. Changes resulting from any comments received were made by the authors on the basis of scientific completeness and accuracy. Susan J. Keam is a contracted employee of Adis International Ltd/Springer Nature, and declares no relevant conflicts of interest. All authors contributed to this article and are responsible for its content. Ethics approval, Consent to participate, Consent to publish, Availability of data and material, Code availability Not applicable. Additional information about this Adis Drug Review can be found here . Abstract Vamorolone (AGAMREE®) is an oral, selective, dissociative corticosteroid developed by ReveraGen BioPharma and Santhera Pharmaceuticals for the treatment of patients with muscular dystrophy. Vamorolone was approved in October 2023 for the treatment of Duchenne muscular dystrophy (DMD) in patients 2 years of age and older in the USA and received a positive opinion in the EU in October 2023 for the treatment of DMD in patients 4 years of age and older. This article summarizes the milestones in the development of vamorolone leading to this first approval for DMD. © Springer Nature Switzerland AG 2023

العلاقة: https://figshare.com/articles/online_resource/Vamorolone_First_Approval/24654435Test

الإتاحة: https://doi.org/10.6084/m9.figshare.24654435.v1Test
https://figshare.com/articles/online_resource/Vamorolone_First_Approval/24654435Test

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

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

الوصف: Duchenne muscular dystrophy (DMD) and spinal muscular atrophy (SMA) are two common and important Inherited neuromuscular disorders which have witnessed immense advances in their treatment owing to ongoing developments in gene therapy. Better modalities for clinical testing and improved clinical awareness has led to facilitation of innovative therapeutic research. Multiple new agents have been approved by regulatory authorities. A continuing research on evaluating such treatment options is required more than ever. These novel therapies have immense potential to transform this field and prolong the functional independence and lifespan of patients.

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

الوصول الحر: https://doaj.org/article/205992cb4c1c4fdf8cda7cc878780529Test

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

الوصف: A white paper addressing the ethical and technical challenges of returning meaningful individual clinical trial results to trial participants

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

المؤلفون: Christina Olsen, Ritchie Head, Jane Knerova, Jane Larkindale, Susan Ward, Pietro Spitali, Hermien Kan

مصطلحات موضوعية: Duchenne muscular dystrophy, Vamorolone, Biomarkers

الوصف: The workshop was held on 29th November 2018 at Motol University Hospital Prague and was a co-run event by the VISION-DMD project and the European Reference Network for Neuromuscular Disease EURO-NMD. The first part of the workshop summarised the state of the art with presentations from leading experts from industry, academia, regulators, patient foundations and the clinical specialists and was attended by over 50 participants. Following the presentations an expert panel discussion with invited participants addressed the current landscape and future aims and objectives. A follow-up discussion (January 2019, Leiden), future planning and summary of biomarker data collated so far are also described.

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

المؤلفون: Olsen, Christina, Head, Ritchie, Knerova, Jane, Larkindale, Jane, Ward, Susan, Spitali, Pietro, Kan, Hermien

مصطلحات موضوعية: Duchenne muscular dystrophy, Vamorolone, Biomarkers

الوصف: The workshop was held on 29th November 2018 at Motol University Hospital Prague and was a co-run event by the VISION-DMD project and the European Reference Network for Neuromuscular Disease EURO-NMD. The first part of the workshop summarised the state of the art with presentations from leading experts from industry, academia, regulators, patient foundations and the clinical specialists and was attended by over 50 participants. Following the presentations an expert panel discussion with invited participants addressed the current landscape and future aims and objectives. A follow-up discussion (January 2019, Leiden), future planning and summary of biomarker data collated so far are also described.

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::2da9a555da5991a9aa9cdbb1ac59a16bTest

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

الوصف: Objective: We carried out first-in-patient studies of vamorolone, a first-in-class dissociative steroidal anti-inflammatory drug, in Duchenne muscular dystrophy. Methods: An open-label, multiple-ascending dose study of vamorolone was conducted in 48 boys with Duchenne muscular dystrophy (4 to <7 years, steroid-naïve). Dose levels were 0.25, 0.75, 2.0 and 6.0 mg/kg/day using an oral suspension formulation (12 boys/dose level; 1/3rd to 10x glucocorticoid dose in DMD). Results: Over a 24-week treatment period, oral administration of vamorolone at all doses tested was safe and well-tolerated. The 2.0 mg/kg/day dose group met the primary efficacy outcome of improved muscle function (time to stand; 24 weeks vamorolone treatment versus natural history controls), without evidence of most adverse effects of glucocorticoids. Significant dose-responsive improvements in 10 meter run/walk, and six-minute walk test were observed for 2.0 and 6.0 mg/kg/day dose groups. The morbidity of most concern to many chronic glucocorticoid users, bone loss, was not seen with vamorolone at any dose, as evidenced by serum osteocalcin. Biomarker outcomes for adrenal suppression and insulin resistance were also less impacted in vamorolone-treated DMD patients, relative to published studies of glucocorticoid therapy. Conclusions: Vamorolone demonstrated both efficacy and a reduction in adverse effects in DMD patients compared to traditional glucocorticoids in a 24-week, open-label study. Vamorolone has potential to replace chronic glucocorticoids in many disorders where side effects detract from patient quality of life. Classification of Evidence: This study provides Class II evidence that certain dosages of vamorolone are well tolerated and effective in improving muscle function in DMD.

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

المؤلفون: Dr Laurie Conklin, Dr Holly Peay, Suzanne Gaglianone, Suzie-Ann Bakker, Elizabeth Vroom, Dr Eric Hoffman, Christina Olsen

مصطلحات موضوعية: education, Duchenne muscular dystrophy, clinical trial, VISION-DMD, return of patient data, vamorolone, 3. Good health

الوصف: A white paper addressing the ethical and technical challenges of returning meaningful individual clinical trial results to trial participants

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_________::cc786295c7cee68c7f66480e0a5f6c77Test

المؤلفون: Dr Laurie Conklin, Dr Holly Peay, Gaglianone, Suzanne, Suzie-Ann Bakker, Vroom, Elizabeth, Dr Eric Hoffman, Olsen, Christina

مصطلحات موضوعية: education, Duchenne muscular dystrophy, clinical trial, VISION-DMD, return of patient data, vamorolone, 3. Good health

الوصف: A white paper addressing the ethical and technical challenges of returning meaningful individual clinical trial results to trial participants

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_________::343ea968da3c7524f4f1378ad65a2100Test