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疾病修饰:有前途的目标和成功的障碍。

Disease modification: promising targets and impediments to success.

机构信息

Orthopaedic & Regenerative Medicine Research, Genzyme, A Sanofi Company, Framingham, MA 01701, USA.

出版信息

Rheum Dis Clin North Am. 2013 Feb;39(1):177-87. doi: 10.1016/j.rdc.2012.10.006.

Abstract

Osteoarthritis (OA) is a significant and growing concern to a large segment of the population. Effective treatments for slowing or stopping the progression of the disease are not available despite a great deal of investment-backed effort on the part of academia, government, and the pharmaceutical industry. Target selection has been problematic. Progress may also have been hindered to some extent by the prevalent cartilage-centric view of OA. Significant clinical development challenges remain for novel therapeutics in this area. This review elaborates on the challenges of disease-modifying OA drug development and points out specific therapeutic intervention strategies recently tried or currently being pursued.

摘要

骨关节炎(OA)是一个重大且日益受到关注的问题,影响着很大一部分人群。尽管学术界、政府和制药行业投入了大量资金,但目前还没有有效的治疗方法来减缓或阻止疾病的进展。目标选择一直存在问题。OA 的普遍软骨中心观点在某种程度上也可能阻碍了进展。在这一领域,新型疗法仍然面临着重大的临床开发挑战。本文详细阐述了改变 OA 疾病进程药物开发的挑战,并指出了最近尝试或正在探索的特定治疗干预策略。

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