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