by Michael Addelman, University of Manchester Credit: Pixabay/CC0 Public Domain Researchers will tell an international conference today (Feb. 24) that an investigational gene therapy for Sanfilippo syndrome—which leads to a form of childhood dementia—has shown promising early results in a proof-of-concept study. It found four out of five patients diagnosed with Sanfilippo have continued to gain cognitive skills...
Tag: <span>childhood dementia</span>
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Enzyme therapy shows promise for childhood dementia
by University of Edinburgh Graphical abstract. Credit: Journal of Clinical Investigation (2022). DOI: 10.1172/JCI163107 Studies into a rare nervous system disorder that causes dementia and early death in children have highlighted a possible treatment for the condition. Regular infusions of a key enzyme that is deficient in children with infantile Batten disease, also called CLN1 disease, led...