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.

Topic · Rare disease ·

delta – Columbus Children's Foundation

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

Save Purnell Today

The legacy of finding a cure for Reno twins Addi and Cassie Hempel

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

Save Purnell Today

March 2022 – NNPDF

NIH clinical trial begins for treatment of rare, fatal neurological disorder

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

Niemann-Pick Type C Disease - NP-C Genetic Disease - Lysosomal Disease