A hopeful therapy for Niemann-Pick C diseases - The Lancet
Por um escritor misterioso
Last updated 20 março 2025


Effects of miglustat therapy on neurological disorder and survival

Potential pharmacological strategies targeting the Niemann-Pick C1

Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological

Effects of miglustat therapy on neurological disorder and survival

Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological

Cyclodextrin Shows Promise in Niemann-Pick Type C Disease

Full article: New therapies in the management of Niemann-Pick type

Catatonia in neurodevelopmental disorders: assessing catatonic

IJMS, Free Full-Text

Clinical applications of gene therapy for rare diseases: A review

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Adult Niemann-Pick disease type C in France: clinical phenotypes

Gene therapy shows promise for treating Niemann-Pick disease type

Niemann–Pick disease, type C - Wikipedia

Niemann–Pick Disease Type C1
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