Accelerate. Develop. Cure.
The Niemann-Pick Type C Therapy Accelerator's mission is to accelerate the development of novel Niemann-Pick Type C therapies and find a cure.
We connect families, researchers, and clinicians with information, tools, data, and funding to advance the development of Niemann-Pick Type C therapies.
The NPC Community welcomes supporters, researchers, companies, and families to get involved with the NPC community. Navigate through this website to learn more.
Neimann-Pick Type C Disease
Niemann-Pick Type C (NPC) disease is a genetic, neurodegenerative disorder which causes progressive deterioration of the nervous system. It usually affects children by interfering with their ability to metabolize cholesterol. Adult onset may also occur. Large amounts of cholesterol accumulate within the liver, spleen, and brain. This metabolic disorder leads to a series of neurological problems that are ultimately fatal…until a treatment is found.
News and Stories
The NPC Therapy Accelerator (NTA) is working hard to forge research collaborations, connect companies to scientists, clinicians and patients, and share a common voice in communication to companies and regulators. Listed below are areas the NTA is helping the NPC community.
Recent Announcements: On August 3rd, members of the NPC Accelerator and the NPC community hosted a 2 hour listening session with the FDA. A detailed report of that meeting can be found below at this link:
May 5, 2020Dear Dr. Woodcock and Dr. Donohue: As leaders across the Neimann-Pick Type C (NPC) Community, we are writing to info...
September 14, 2020NTA to work with Companies & NIH to have the FDA evaluate Miglustat as an indication for NPC diseaseMiglustat improves swallowing in children and adolescents with Niemann-Pick type C1 disease.
January 15, 2021On behalf of the Niemann-Pick Type C Therapy Accelerator (NTA) team, we would like to share with you a recent patient a...
February 18, 2021Dear Steve and Elissa, I am writing on behalf of the Niemann-Pick Type C (NPC) Therapy Accelerator in regard to the ...
May 25, 2021Sean Kassen, APMRFJustin Hopkin, NNPDFPhil Marella, Danas Angels Research Trust, NPC ParentCristin Davidson, NPC Sc...
June 2, 2021AttendeesSean KassenJoslyn CrowePhil MarellaChris AndrewsCristin DavidsonDave Zook Topics- Mandos acquisition of...
August 26, 2021August 13, 2021 Kathleen Donohue, MD Food and Drug Administration Center for Drug Evaluation and Research Offi...
September 20, 2021On August 3, 2021, the Ara Parseghian Medical Research Fund (APMRF), International Niemann-Pick Disease Alliance (INPDA...