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Stargardt macular dystrophy and evolving therapies

21 Aug 2018

Abstract excerpt

INTRODUCTION: Stargardt macular dystrophy (STGD1) is a hereditary retinal degeneration that lacks effective treatment options. Gene therapy, stem cell therapy, and pharmacotherapy with visual cycle modulators (VCMs) and complement inhibitors are discussed as potential treatments. AREAS COVERED: Investigational therapies for STGD1 aim to reduce toxic bisretinoids and lipofuscin in the retina and retinal pigment...

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PMID: 30129371
DOI: 10.1080/14712598.2018.1513486

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