Advances in the treatment of neuronal ceroid lipofuscinosis
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DOI: 10.1080/21678707.2019.1684258
Abstract: ABSTRACT Introduction: Neuronal ceroid lipofuscinoses (NCL) are neurodegenerative lysosomal storage disorders typically characterized by cognitive and visual impairments, epileptic seizures, ataxia, and deterioration of motor skills. Recent success of Brineura® for the treatment of neurologic… read more here.
Keywords: treatment neuronal; neuronal ceroid; treatment; ceroid lipofuscinosis ... See more keywords