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- W252656299 endingPage "2300" @default.
- W252656299 startingPage "2292" @default.
- W252656299 abstract "The neuronal ceroid lipofuscinoses represent a group of severe childhood lysosomal storage diseases. With at least 13 identified variants they are the most common cause of inherited neurodegeneration in children. These diseases share common pathological characteristics including motor problems, vision loss, seizures, and cognitive decline, culminating in premature death. Currently, no form of the disease can be treated or cured, with only palliative care to minimise discomfort. This review focuses on current and potentially ground-breaking clinical trials, including small molecule, enzyme replacement, stem cell, and gene therapies, in the development of effective treatments for the various disease subtypes. This article is part of a Special Issue entitled: Current Research on the Neuronal Ceroid Lipofuscinoses (Batten Disease)." @default.
- W252656299 created "2016-06-24" @default.
- W252656299 creator A5022030316 @default.
- W252656299 creator A5038401925 @default.
- W252656299 creator A5043382019 @default.
- W252656299 creator A5047605160 @default.
- W252656299 date "2015-10-01" @default.
- W252656299 modified "2023-09-27" @default.
- W252656299 title "Experimental therapies in the neuronal ceroid lipofuscinoses" @default.
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