A hopeful therapy for Niemann-Pick C diseases - The Lancet
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Last updated 26 julho 2024
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Full article: New therapies in the management of Niemann-Pick type
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Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological
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PDF) Consensus clinical management guidelines for Niemann-Pick
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Improved systemic AAV gene therapy with a neurotrophic capsid in
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Effects of miglustat therapy on neurological disorder and survival
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Polyrotaxane-based systemic delivery of β-cyclodextrins for
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A hopeful therapy for Niemann-Pick C diseases - The Lancet
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Lysosomal storage disorders: from biology to the clinic with
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Neurological manifestations of lysosomal disorders and emerging
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