dc.creatorConran, Nicola
dc.date2015-Feb
dc.date2015-11-27T13:46:09Z
dc.date2015-11-27T13:46:09Z
dc.date.accessioned2018-03-29T01:23:33Z
dc.date.available2018-03-29T01:23:33Z
dc.identifierExpert Opinion On Investigational Drugs. , p. 1-8, 2015-Feb.
dc.identifier1744-7658
dc.identifier10.1517/13543784.2015.1012292
dc.identifierhttp://www.ncbi.nlm.nih.gov/pubmed/25682977
dc.identifierhttp://repositorio.unicamp.br/jspui/handle/REPOSIP/202125
dc.identifier25682977
dc.identifier.urihttp://repositorioslatinoamericanos.uchile.cl/handle/2250/1302358
dc.descriptionSickle cell disease (SCD) is a genetic disorder characterized by the production of abnormal hemoglobin that polymerizes at low oxygen concentrations, causing the erythrocyte to adopt a sickle-shaped morphology. SCD pathophysiology is extremely complex and can lead to numerous clinical complications, including painful vaso-occlusive crises (VOC), end-organ damage, and a shortened lifespan. An impressive number of investigational drugs are currently in early stages of clinical development with prospects for use either as chronic therapies to reduce VOC frequency and end-organ damage in SCD or for use at the time of VOC onset. Many of these agents have been developed using a pathophysiological-based approach to SCD, targeting one or more of the mechanisms that contribute to the disease process. It is plausible that a multi-drug approach to treating the disease will evolve in the coming years, whereby hydroxyurea (HU) (the only drug currently FDA-approved for SCD) is used in combination with drugs that amplify nitric oxide signaling and/or counteract hemolytic effects, platelet activation and inflammation.
dc.description
dc.description1-8
dc.languageeng
dc.relationExpert Opinion On Investigational Drugs
dc.relationExpert Opin Investig Drugs
dc.rightsfechado
dc.rights
dc.sourcePubMed
dc.subjectEarly Investigational Drugs
dc.subjectPathophysiology
dc.subjectSickle Cell Disease
dc.subjectVaso-occlusion
dc.titleProspects For Early Investigational Therapies For Sickle Cell Disease.
dc.typeArtículos de revistas


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