By Cliff Djajapranata The Ara Parseghian Medical Research Fund (APMRF) at Notre Dame and its collaborators hosted a patient-focused drug development (PFDD) meeting on Niemann-Pick Type C (NPC) on March 18 to bring together those affected by the disease, the Food and Drug Administration, drug developers, and other stakeholders. The PFDD meeting, held in Hyattsville, Maryland, provided persons with NPC … Read More

Dear Friends in the NPC Community, The Ara Parseghian Medical Research Fund is excited to announce that, in collaboration with the organizations listed below, on March 18th, 2019 we will host the Niemann-Pick Type C (NPC) externally-led Patient-Focused Drug Development (PFDD) meeting with the FDA. What is the externally led Patient-Focused Drug Development (PFDD) meeting? The PFDD meeting is a groundbreaking initiative driven by persons … Read More

Dear Friends in the NPC Community, The Ara Parseghian Medical Research Fund is excited to announce that, in collaboration with the organizations listed below, on March 18th, 2019 we will host the Niemann-Pick Type C (NPC) externally-led Patient-Focused Drug Development (PFDD) meeting with the FDA. What is the externally led Patient-Focused Drug Development (PFDD) meeting? The PFDD meeting is a … Read More

Dear members of the NPC community, Please view this video from Steve Romano, the Chief Scientific Officer at Mallinckrodt Pharmaceuticals, discussing the VTS-270 clinical trial and reconfirming Mallinckrodt’s commitment to the NPC community. https://players.brightcove.net/2558692123001/default_default/index.html?videoId=5973271711001  

Dear Friends in the NPC Community, The Ara Parseghian Medical Research Fund is excited to announce that, in collaboration with the organizations listed below, we will host the Niemann-Pick Type C (NPC) externally-led Patient-Focused Drug Development (PFDD) meeting. In late August, we submitted a Letter of Intent to the Food and Drug Administration (FDA) for approval to conduct this landmark … Read More

For the past six years, the University of Notre Dame Men’s Rugby team has played the University of Arizona in an annual match called the Parseghian Cup. The team’s come together each year do the relationship the Parseghian family has with the Notre Dame family and the Tucson, AZ community (home to the Arizona Wildcats). The universities take turns hosting the event, and … Read More

Thank you to CNN for dedicating an entire episode of their series Something’s Killing Me toward investigating and unpacking NPC, telling the story of many NPC families and their fight to find a cure for this devastating disease. It aired on Sunday, August 19 at 8pm on the Headline News Network. We invite you to watch this impactful episode that … Read More

On June 2-5 of this year, over 140 NPC researchers, clinicians, and family members gathered in Tucson, AZ for our annual Conference.  The level of participation and collaboration was incredible during our time together. The conference kicked off with a keynote address by Dr. Thijn Brummelkamp, who spoke on genetic studies on the function of NPC1 in pathogen entry and cholesterol … Read More

I am excited to announce the Together Strong NPC Foundation will be accepting grant requests.  The foundation was set up with three primary goals listed below: 1) NPC Science:  Develop foundational science to benefit NPC patients; advance research and development projects that focus on discovery, identification of therapeutic targets, and improved diagnostics 2) Public Awareness and Advocacy:  Enable stronger independent … Read More

I am please to announce this year’s Parseghian Fund grantees.  Thanks to the generosity of so many and the commitment of these scientists and clinicians the APMRF was able to fund 14 new projects that will help find a cure for NPC disease.  The research projects listed below represent a variety of important areas including furthering our understanding of NPC … Read More