Niemann-Pick Disease Type C

News

Investors

Careers

Contacts

Adult living with Niemann-Pick disease type C

Learn More

Niemann-Pick disease type C

People with NPC have changes in their lysosomal function impacting their body’s ability to appropriately transport cholesterol and other lipids within their cells. Over time, this accumulation causes progressive dysfunction of the nerves, brain, and other organs, resulting in neurodegenerative and visceral symptoms. These symptoms include negative impacts on speech, cognitive function, fine motor skills, and the ability to swallow.

NPC is an inherited disorder that can affect patients at any age. The symptoms for NPC are variable across patients, and many people with NPC experience a prolonged period before diagnosis due to the lack of disease awareness. Disease progression is irreversible in all patients and varies by the age of symptom onset.

Resources

Ara Parseghian Medical Research Fund – Fighting for Cure for Niemann-Pick Type C Disease Hope for Marian International Niemann–Pick Disease Alliance International Niemann-Pick Disease Registry National Niemann-Pick Disease Foundation Niemann-Pick UK Shining a Light on NPC – Firefly Fund

Adult living with Niemann-Pick disease type c