ND Expert Sean Kassen: Statement on first FDA-approved treatment for Niemann-Pick Type C disease
Today, the FDA announced the first approved treatment for Niemann-Pick Type C (NPC) disease: an oral medication named Miplyffa (arimoclomol). Sean Kassen, director of the Ara Parseghian Medical Research Fund at the University of Notre Dame, said this represents the beginning of a new and hopeful era for NPC families.
“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 advance our understanding of NPC and bring treatments to those affected by this devastating disease. Today marks a momentous occasion with the FDA approval of Miplyffa and the start of a new era with the first official medication for the treatment of NPC disease. We will continue to fight for NPC families until this disease is eradicated,” Kassen said.
The APMRF is a non-profit organization dedicated to finding a treatment or cure for Niemann-Pick Type C disease. The organization is named in honor of Ara Parseghian, the much beloved and well-known Notre Dame football coach, and was founded by Mike and Cindy Parseghian, whose three children — Michael, Marcia and Christa — were diagnosed with NPC in 1994.
Latest ND NewsWire
- Former Secretary of State Condoleezza Rice to speak at Notre Dame Forum eventCondoleezza Rice, the 66th U.S. Secretary of State, the Tad and Dianne Taube Director of the Hoover Institution and a Notre Dame alumna, will participate in a “fireside chat” with University President Rev. Robert A. Dowd, C.S.C., at 4 p.m. Friday (Oct. 11) as part of the 2024-25 Notre Dame Forum. The conversation, titled “The Perils of U.S. Isolationism,” will take place in the Leighton Concert Hall in the DeBartolo Performing Arts Center and will also be livestreamed. The event is free, but ticketed, with tickets available on a first-come, first-served basis beginning one hour beforehand.
- Political scientist explores extending constitutional duties to private actorsNew research from Christina Bambrick, the Filip Family Assistant Professor of Political Science at Notre Dame, explores the nonconventional idea that each of us, as private citizens, may be responsible for upholding the constitutional rights of our fellow citizens. She examines constitutional politics across the globe to explore these different approaches to balancing rights and responsibilities in a democratic society.
- Alumni Association honors outstanding alumni during fall board of directors meetingThe University of Notre Dame Alumni…
- Six new faculty join psychology department to advance research on mental health, other disordersThe Department of Psychology at the University of Notre Dame has hired six new faculty members this year, a significant expansion of a field that is core to the University’s commitment to fighting the U.S. mental health crisis.
- To prevent opioid misuse in teens and young adults, researchers turn to AI and social media dataResearchers at Notre Dame are developing a new artificial intelligence-driven paradigm built on social media data to provide insights into personalized interventions for reducing opioid misuse and death among teens and young adults.
- Notre Dame receives significant gift from the Ernestine Morris Carmichael Raclin estateThe Raclin Murphy Museum of Art at the University of Notre Dame is the beneficiary of a significant gift of paintings, sculptures, and decorative art objects from the estate of Ernestine Morris Carmichael Raclin.