International Niemann–Pick Disease Alliance

Trials: A Desperate Fight to Save Kids & Change Science

For six years, The Wall Street Journal followed a group of parents and scientists seeking a treatment for a rare and fatal genetic disease that strikes primarily children. Their collaboration accelerated development of a promising drug and, along the way, pushed the boundaries of medical research itself.

Follow this link to learn more about the story associated with Niemann-Pick Disease Type C:

Join the Registry

Calling all patients and carers! We've created a global registry of all NPD patients - if you're not enrolled yet, join us now...

The Impact of NPC

The global community tell their stories...