2019 Parseghian Fund Cocktail Reception

On Friday, October 11, 2019, friends of the APMRF gathered on Notre Dame’s campus to spend time together, and learn about the Parseghian’s fight to find a cure for Niemann-Pick Type C (NPC) disease. The Parseghian’s were also joined by Cheryl and Paul Marcogliese and their two boys, Daniel and James. Special thank you to Cheryl who talked to the … Read More

Letter from Mallinckrodt concerning the VTS-301 trial in the UK

Dear Toni and Niemann-Pick UK Members, Good news!  We are pleased to inform you that Mallinckrodt has confirmed with the Medicines and Healthcare Products Regulatory Agency (MHRA) that the temporary halt of Study VTS301 Part C has been lifted and the study may resume in the United Kingdom.  Mallinckrodt is working with the MHRA and the study investigators to ensure … Read More

September update from Mallinckrodt concerning VTS-270 in the UK and France

As we continue to receive questions about the recent suspension of the open label extension portion (Part C) of our Phase 2b/3 study in the UK and France, I wanted to reach out to share another brief update based on the information we have available at this time. We understand that there may be a desire for some study participants … Read More

Mallinckrodt update concerning VTS-270 in the UK and France

On Wednesday, July 31, 2019 Mallinckrodt was notified by the United Kingdom (UK) Medicines and Healthcare products Regulatory Agency and the French Agence Nationale de Sécurité du Médicament that the open label extension portion (Part C) of our Phase 2b/3 study VTS301, evaluating the effects of adrabetadex (VTS-270) in children who have neurologic symptoms of Niemann-Pick Type C (NPC) disease, … Read More

2019 APMRF Grant Awardees

Researchers at Notre Dame

The APMRF is excited to announce the 2019 Grantees. Thanks to the generosity of so many, we were able to support 15 new grants that will help us find a cure for NPC disease. These high-impact research projects represent a variety of important areas including furthering our understanding of NPC biology, identifying novel biomarkers, improving and developing new therapeutics, and … Read More

APMRF and partners host meeting to hear from those affected by NPC disease

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

REGISTER NOW — Niemann-Pick Type C (NPC) externally-led Patient-Focused Drug Development (PFDD) meeting

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

Niemann-Pick Type C externally-led Patient-Focused Drug Development (PFDD) meeting and pre-meeting survey

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

Video update from Mallinckrodt in regards to the VTS-270 Clinical Trial

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  

Niemann-Pick Type C Externally Led Patient-Focused Drug Development Meeting Announcement

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