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

Children with Niemann-Pick Type C (NPC), their parents and scientists seeking a treatment for this fatal genetic disease. WSJ follows this 6-year fight.

Families plead with FDA for drug to treat Niemann-Pick Disease Type C

Twins with rare, fatal disease at center of multimillion-dollar lawsuit

Niemann–Pick type C disease: cellular pathology and pharmacotherapy - Wheeler - 2020 - Journal of Neurochemistry - Wiley Online Library

Niemann-Pick disease type C as a neurovisceral disease. Schematic

March 2022 – NNPDF

Frontiers Case Report: Be Aware of “New” Features of Niemann–Pick Disease: Insights From Two Pediatric Cases

Experimental treatment helps toddler with rare genetic disease - CBS News

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

delta – Columbus Children's Foundation

Baby With Niemann-Pick Disease — Marian McGlocklin Story

Experimental drug shows promise in cruel childhood disease

Morbidity and mortality in type B Niemann–Pick disease

Parents of kids with Niemann Pick C advocate for adrabetadex

Frontiers Case Report: Be Aware of “New” Features of Niemann–Pick Disease: Insights From Two Pediatric Cases