NPC Listening Session with the FDA on August 3rd – Final Report

On August 3, 2021, the Ara Parseghian Medical Research Fund (APMRF), International Niemann-Pick Disease Alliance (INPDA), and National Niemann-Pick Disease Foundation (NNPDF) convened a listening session with representatives from the U.S. Food and Drug Administration (FDA) Center for Drug Evaluation and Review (CDER) Office of New Drugs (OND). This session, which was held at the request of the FDA, gave … Read More

Pfreigers Digests – September 2021

Research at Notre Dame

Update from Mandos, LLC

Mandos is a biopharmaceutical company committed to furthering development of adrabetadex* for Niemann-Pick Type C. The company is led by a diverse and passionate management team experienced in every stage of the drug development process from research through commercialization. Please find a link to the September communication from Mandos, LLC.

Letter from the NPC community following the FDA Listening Session on August 3, 2021

August 13, 2021 Kathleen Donohue, MD Food and Drug Administration Center for Drug Evaluation and Research Office of New Drugs Division of Rare Diseases and Medical Genetics 10903 New Hampshire Avenue Silver Spring, MD 20993 RE: August 3, 2021 FDA-NPC Community Listening Session Dear Dr. Donohue: On behalf of the Ara Parseghian Medical Research Fund, the National Niemann-Pick Disease Foundation, … Read More

Niemann-Pick Type C Caregiver Preference Survey on Benefit Risk for Potential Treatments

Dear Friends in the NPC Community, We invite families or caregivers of persons with Niemann-pick Type C disease to take a new survey that is designed to collect information about your priorities and preferences for treating Niemann-Pick Type C. It is organized by the NPC Therapy Accelerator*.  In the survey, you will read about hypothetical treatments for Niemann-Pick Type C.  … Read More

Positive results reported from IntraBio’s multinational clinical trial with IB1001 for treatment of Niemann-Pick disease Type C

A multinational clinical trial of IB1001 for the treatment of Niemann-Pick disease Type C (NPC) demonstrated a statistically significant and clinically meaningful improvement in symptoms, functioning, and quality of life in both primary and topline secondary endpoints for both pediatric and adult patients with NPC. The trial met its primary endpoint, the Clinical Impression of Change in Severity (CI-CS), which was … Read More

2020 Michael, Marcia and Christa Parseghian Scientific Conference for Niemann-Pick Type C Research Conference- Virtual Symposiums

This past year, the annual in-person conference in Tucson, AZ was cancelled due to the COVID-19 / novel Coronavirus pandemic. Because sharing research progress is essential, we developed a new format in place of the Tucson conference. We hosted a series of virtual symposia throughout the month of May and June to provide key research updates from APMRF-funded researchers and invited speakers, … 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

2017 APMRF Funded Grants

We are excited to announce the 2017 APMRF funded grants.  Please go to this LINK to see the list of funded researchers and the focus of their research. Research areas range from fundamental science of the NPC pathway, the development of new therapeutics for NPC disease, and also the support of new gene-therapy efforts.  We are also very excited that … Read More