Clinical challenges and future therapeutic approaches for neuronal ceroid lipofuscinosis
Abstract
Treatment of the neuronal ceroid lipofuscinoses, also known as Batten disease, is at the start of a new era because of diagnostic and therapeutic advances relevant to this group of inherited neurodegenerative and life-limiting disorders that affect children. Diagnosis has improved with the use of comprehensive DNA-based tests that simultaneously screen for many genes. The identification of disease-causing mutations in 13 genes provides a basis...
Paper Details
Title
Clinical challenges and future therapeutic approaches for neuronal ceroid lipofuscinosis
Published Date
Jan 1, 2019
Journal
Volume
18
Issue
1
Pages
107 - 116
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