dc.creator | Conran, Nicola | |
dc.date | 2015-Feb | |
dc.date | 2015-11-27T13:46:09Z | |
dc.date | 2015-11-27T13:46:09Z | |
dc.date.accessioned | 2018-03-29T01:23:33Z | |
dc.date.available | 2018-03-29T01:23:33Z | |
dc.identifier | Expert Opinion On Investigational Drugs. , p. 1-8, 2015-Feb. | |
dc.identifier | 1744-7658 | |
dc.identifier | 10.1517/13543784.2015.1012292 | |
dc.identifier | http://www.ncbi.nlm.nih.gov/pubmed/25682977 | |
dc.identifier | http://repositorio.unicamp.br/jspui/handle/REPOSIP/202125 | |
dc.identifier | 25682977 | |
dc.identifier.uri | http://repositorioslatinoamericanos.uchile.cl/handle/2250/1302358 | |
dc.description | Sickle 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.description | 1-8 | |
dc.language | eng | |
dc.relation | Expert Opinion On Investigational Drugs | |
dc.relation | Expert Opin Investig Drugs | |
dc.rights | fechado | |
dc.rights | | |
dc.source | PubMed | |
dc.subject | Early Investigational Drugs | |
dc.subject | Pathophysiology | |
dc.subject | Sickle Cell Disease | |
dc.subject | Vaso-occlusion | |
dc.title | Prospects For Early Investigational Therapies For Sickle Cell Disease. | |
dc.type | Artículos de revistas | |