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Type: Artigo de periódico
Title: Prospects For Early Investigational Therapies For Sickle Cell Disease.
Author: Conran, Nicola
Abstract: 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.
Subject: Early Investigational Drugs
Sickle Cell Disease
Citation: Expert Opinion On Investigational Drugs. , p. 1-8, 2015-Feb.
Rights: fechado
Identifier DOI: 10.1517/13543784.2015.1012292
Date Issue: 2015
Appears in Collections:Unicamp - Artigos e Outros Documentos

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