Treating Niemann-Pick Disease Type C

Niemann-Pick Type C Disease Reveals a Link between Lysosomal Cholesterol and PtdIns(4,5)P2 That Regulates Neuronal Excitability - ScienceDirect

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

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

Therapeutic intervention for Niemann–Pick type C disease must target

What You See and What You Don't: Diagnosing Niemann Pick, Type C - Complex Child

Niemann–Pick type C Mnemonic

Walgreen's Support Allows Our Twins with Niemann Pick Type C To Receive Cyclodextrin Treatments At Home

Terapia Génica para Niemann Pick tipo C - Grupo Teaming

NNPDF on X: #NNPDF partnered with @RareDiseases to create this animated educational resource on Niemann Pick Type C. Please help us raise awareness of #NPC by sharing this video with your networks

Meet Parker and Woodrow, two AZ children with a rare disease