The U.S. Food and Drug Administration approved the first treatment for Niemann-Pick disease type C. “It has been almost 30 years to the day since Michael, Marcia and Christa Parseghian were diagnosed with Niemann-Pick Type C disease, eventually taking their young lives. Since that time the Parseghian family and so many other families, researchers and volunteers have worked tirelessly to … Read More
Dr. Ross Fredenburg to oversee a new initiative focused on identifying new therapies to correct mutant NPC1 protein folding and trafficking.
In 2022, the APMRF received a $1 million gift from Steve and Maureen Klinsky , along with additional donations from the annual Parseghian Classic, to launch a new program focusing on identifying new therapies for Niemann Pick Type C patients. On November 18th, a volunteer group of NPC scientists and clinicians, along with industry experts, gathered in Chicago for a special … Read More
Duke-Margolis NPC Endpoint Considerations Workshop Summary Now Available
Please find the workshop summary from the Duke-Margolis NPC Endpoint Considerations Workshop is now available on the Duke-Margolis website and attached to this email. They will also be holding a brief webinar along with the FDA on Thursday, August 4, from 3:00-4:00 pm ET to provide an overview of the key themes and future directions shared during the January 2022 workshop and presented in this newly released workshop … Read More
Letter from the NPC community to Dr. McClellan, director of the Duke-Margolis Center
On Monday, December 13th, members of the NPC community sent a letter to Dr. McClellan, who is the director of the Duke-Margolis Center that is hosting a workshop on NPC endpoints on January 24 & 25. The letter and response is below. It is imperative to the NPC community that the NPC- Clinical Severity Scale is a key topic of … Read More
Parseghian Fund Fall Fundraiser and Reception
Parseghian Fund Fall Reception – 2021On Friday, October 1, the Parseghian Fund’s hosted their annual Fall fundraiser and reception. It was so great to be able to see and spend time with everyone…in person! We were also grateful that attendees were able to hear from Cindy Parseghian, Santiago Schnell, and Samantha Berns. Samantha is battling NPC disease and was able … Read More
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
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. https://mandoshealth.com/communications.html
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
Orphazyme announces publication of results from its Phase 2/3 trial of arimoclomol in Niemann-Pick disease type C in the Journal of Inherited Metabolic Disease
Orphazyme A/S today announced that results from a Phase 2/3 trial of arimoclomol, an investigational heat-shock protein amplifier, in Niemann-Pick disease type C (NPC) have been published in the peer-reviewed Journal of Inherited Metabolic Disease (JIMD). You can find a link to the press release here and click here for the LINK to the publication.