A review and update on orphan drugs for the treatment of noninfectious uveitis

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A review and update on orphan drugs for the treatment of noninfectious uveitis

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dc.contributor.author You, Caiyun en_US
dc.contributor.author Sahawneh, Haitham F en_US
dc.contributor.author Ma, Lina en_US
dc.contributor.author Kubaisi, Buraa en_US
dc.contributor.author Schmidt, Alexander en_US
dc.contributor.author Foster, C Stephen en_US
dc.date.accessioned 2017-03-28T23:48:05Z
dc.date.issued 2017 en_US
dc.identifier.citation You, Caiyun, Haitham F Sahawneh, Lina Ma, Buraa Kubaisi, Alexander Schmidt, and C Stephen Foster. 2017. “A review and update on orphan drugs for the treatment of noninfectious uveitis.” Clinical Ophthalmology (Auckland, N.Z.) 11 (1): 257-265. doi:10.2147/OPTH.S121734. http://dx.doi.org/10.2147/OPTH.S121734. en
dc.identifier.issn en
dc.identifier.uri http://nrs.harvard.edu/urn-3:HUL.InstRepos:31731652
dc.description.abstract Introduction: Uveitis, a leading cause of preventable blindness around the world, is a critically underserved disease in regard to the medications approved for use. Multiple immunomodulatory therapy (IMT) drugs are appropriate for uveitis therapy but are still off-label. These IMT agents, including antimetabolites, calcineurin inhibitors, alkylating agents, and biologic agents, have been designated as “orphan drugs” and are widely used for systemic autoimmune diseases or organ transplantation. Area covered The purpose of this paper is to comprehensively review and summarize the approved orphan drugs and biologics that are being used to treat systemic diseases and to discuss drugs that have not yet received approval as an “orphan drug for treating uveitis” by the US Food and Drug Administration (FDA). Our perspective IMT, as a steroid-sparing agent for uveitis patients, has shown promising clinical results. Refractory and recurrent uveitis requires combination IMT agents. IMT is continued for a period of 2 years while the patient is in remission before considering tapering medication. Our current goals include developing further assessments regarding the efficacy, optimal dose, and safety in efforts to achieve FDA approval for “on-label” use of current IMT agents and biologics more quickly and to facilitate insurance coverage and expand access to the products for this orphan disease. en
dc.language.iso en_US en
dc.publisher Dove Medical Press en
dc.relation.isversionof doi:10.2147/OPTH.S121734 en
dc.relation.hasversion http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5298311/pdf/ en
dash.license LAA en_US
dc.subject immunomodulatory en
dc.subject orphan drug en
dc.subject steroid sparing en
dc.subject uveitis en
dc.title A review and update on orphan drugs for the treatment of noninfectious uveitis en
dc.type Journal Article en_US
dc.description.version Version of Record en
dc.relation.journal Clinical Ophthalmology (Auckland, N.Z.) en
dash.depositing.author Foster, C Stephen en_US
dc.date.available 2017-03-28T23:48:05Z

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