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.

When Drug Development for Rare Disease Hit Setback, Parents Were Stung - WSJ

Understanding the phenotypic variability in Niemann-Pick disease type C (NPC): a need for precision medicine

Niemann–Pick disease, type C - Wikipedia

Niemann-Pick disease type C, Orphanet Journal of Rare Diseases

Mother Alleges Biotech Firm Stole Data From Her Sick Children

IJMS, Free Full-Text

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

Gene therapy shows promise for treating Niemann-Pick disease type C1

Lipid trafficking defects in Niemann-Pick type C disease

We know it works': Families, doctor fight move to discontinue medication