Niemann–Pick Disease Type C

Niemann-Pick disease type C (NPC) is a disabling, lysosomal-storage disorder that has been diagnosed prenatally, neonatally, during childhood, and even into adulthood.

Niemann-Pick Disease Type C – EffRx

Niemann-Pick disease types A and B (NORD): Video

PDF] Niemann-Pick Type C Disease—Pathophysiology and Future Perspectives for Treatment

Patient with Niemann-Pick disease type C: over 20 years' follow-up

Niemann-Pick disease type C symptomatology: an expert-based clinical description, Orphanet Journal of Rare Diseases

Potential treatment for Niemann-Pick type C, a rare neurodegenerative disease

Proposed pathological cascade in Niemann-Pick disease type C (NPC)

Immune dysfunction in Niemann‐Pick disease type C - Platt - 2016 - Journal of Neurochemistry - Wiley Online Library

The pathogenesis of Niemann–Pick type C disease: a role for autophagy?, Expert Reviews in Molecular Medicine

Miglustat in Niemann-Pick disease type C patients: a review, Orphanet Journal of Rare Diseases

Niemann–Pick disease type C1 (NPC1) is associated with early-onset

NNPDF on X: October is Global Niemann-Pick Disease Awareness Month! For more information on Niemann-Pick Disease or to make a donation to NNPDF go to #niemannpick #ASMD #NPC #raredisease #NNPDF #NiemannPickC

mRNA Treatment Rescues Niemann–Pick Disease Type C1 in Patient Fibroblasts