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.

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

Save Purnell Today

delta – Columbus Children's Foundation

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

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

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

Parents of twins with Niemann-Pick find a treatment

Niemann Pick Disease - Rivin

Reno Twins With Rare Disease Involved in Huge Lawsuit - O'Mara Law

PDF) Recent Advances in the Diagnosis and Treatment of Niemann-Pick Disease Type C in Children: A Guide to Early Diagnosis for the General Pediatrician

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

Niemann-Pick Disease, Type a disease: Malacards - Research Articles, Drugs, Genes, Clinical Trials

IJMS, Free Full-Text

Topic · Rare disease ·