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.

Donate to cure Niemann Pick disease and save lives!, Jennifer Mitchell posted on the topic

Mother Alleges Biotech Firm Stole Data From Her Sick Children

Efficacy and safety of arimoclomol in Niemann‐Pick disease type C: Results from a double‐blind, randomised, placebo‐controlled, multinational phase 2/3 trial of a novel treatment - Mengel - 2021 - Journal of Inherited

delta – Columbus Children's Foundation

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

Rare Disease Video - Niemann-Pick Disease Types A & B - National Organization for Rare Disorders

Tolerance of chronic HDACi treatment for neurological, visceral and lung Niemann-Pick Type C disease in mice

Parents of twins with Niemann-Pick find a treatment

IJMS, Free Full-Text

Parents of kids with Niemann Pick C advocate for adrabetadex

Two-Year-Old Kamryn Living with Niemann-Pick Disease Type C (NPC) - The Children's Inn at NIH

Save Purnell Today

Push to spur drugs for deadly rare diseases

For a Rare Disease, Drug Trials Scramble for Patients - WSJ

Ohio 2-year-old battling rare Niemann-Pick disease, dubbed 'childhood Alzheimer's