International Niemann–Pick Disease Alliance
Myelin Defects in Niemann–Pick Type C Disease: Mechanisms and Possible Therapeutic Perspectives
Improved systemic AAV gene therapy with a neurotrophic capsid in Niemann-Pick disease type C1 mice
Think NP-C. Talk NP-C – Ataxia
Think NP-C. Talk NP-C – Dysarthria
Think NP-C. Talk NP-C – Gelastic Cataplexy
Think NP-C. Talk NP-C – Hepatosplenomegaly
Think NP-C. Talk NP-C – Organic Psychosis
Think NP-C. Talk NP-C – Vertical Supranuclear Gaze Palsy
Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases
neurological disease progression in Niemann-Pick disease,
type C1: a non-randomised, open-label, phase 1–2 trial
Think Again. Think NP-C Educational Slide Deck
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