Kebilidi is the first gene therapy approved by the FDA for administration directly into the brain, given via a cannula during a surgical procedure, and replaces the human dopa decarboxylase (DDC ...

The arrows/enzymes in red are the enzymes affected in cases of dopa-responsive dystonia. Dashed lines indicate that there are multiple steps in the reaction path. AADC = aromatic L-amino acid ...

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The table below is a review of notable updates that occurred in May 2025 for investigational products in development (not an inclusive list). Click on the status to view our full coverage.

IN 1959 Schayer reported that when rat skin was treated with compound 48/80 the histidine decarboxylase activity increased 1.In later investigations he found that a similar effect was produced by ...

（编辑推荐）本综述系统阐述了L-多巴（L-DOPA）作为多巴胺直接前体，通过酪氨酸羟化酶（TH）和芳香族L-氨基酸脱羧酶（AADC）的合成路径，及其跨越血脑屏障（BBB）治疗帕金森病（PD）的核心机制。文章拓展了其在阿尔茨海默病（AD）、亨廷顿病（HD）等神经退行 ...

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Background: Dopa responsive dystonia (DRD) is a disorder characterised by childhood onset dystonia but a wide range of clinical presentations has now been described. Objective: To study a large ...