ND Expert Sean Kassen: Statement on first FDA approved treatment for Niemann Pick Type C disease

Published:September 20, 2024

Author:Brandi Wampler

Sean Kassen, director of the Ara Parseghian Medical Research Fund at Notre Dame.

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.

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