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Current Drug and Molecular Therapies for the Treatment of Atrophic Age-Related Macular Degeneration: Phase I to Phase III Clinical Development.

EXPERT OPINION ON INVESTIGATIONAL DRUGS(2017)

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摘要
Introduction: Age-related macular degeneration (AMD) is the leading cause of vision loss among the elderly. Atrophic AMD, including early, intermediate and geographic atrophy (GA), accounts for similar to 90% of all cases. It is a multifactorial degeneration characterized by chronic inflammation, oxidative stress and aging components. Although no FDA-approved treatment yet exists for the late stage of atrophic AMD, multiple pathological mechanisms are partially known and several promising therapies are in various stages of development. Areas covered: Underlying mechanisms that define atrophic AMD will help provide novel therapeutic targets that will address this largely unmet clinical need. The purpose of this paper is to review current promising drugs that are being evaluated in clinical trials. Because no pharmacological treatments are currently available for late stage of atrophic AMD, any new therapy would have extensive market potential. Expert opinion: The number of AMD patients is predicted to increase to similar to 30 million worldwide by 2020. In response to this enormous unmet clinical need, new promising therapies are being developed and evaluated in clinical trials. We propose that the assessment of novel interventions will also need to consider the genotypes of participants, as the benefit may be determined by polymorphisms in an individual's genetic background.
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关键词
Atrophic age-related macular degeneration,geographic atrophy,complement,inflammation,visual cycle modulators,antioxidants,toxic by-products,vascular enhancers,stem cell therapy,gene therapy
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