TRIALS: A Fight for Children with Niemann-Pick Type C, a Fatal Genetic Disease

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

Rare Diseases Research: Clinical Trial for Niemann-Pick Type C

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

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

Terapia Génica para Niemann Pick tipo C - Grupo Teaming

Elevation of plasma lysosphingomyelin-509 and urinary bile acid metabolite in Niemann-Pick disease type C-affected individuals - Top Articles selected by the Editor - Molecular Genetics and Metabolism Reports - Journal - Elsevier

Niemann–Pick disease, type C - Wikipedia

Niemann-Pick type C disease is associated with mtDNA disorganization.

Science Update: Biomarker for Niemann-Pick type C may be useful for diagnosing other class of rare disorders, NIH study suggests

Doença de Niemann-Pick – Wikipédia, a enciclopédia livre