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
- Notre Dame mourns the passing of President Jimmy CarterThe University of Notre Dame’s president, Rev. Robert A. Dowd, C.S.C., joined today with leaders worldwide in mourning the death of former President Jimmy Carter at age 100 at his home in Plains, Georgia.
- Robinson Center awarded Early Learning Indiana grant to expand preschool, boost teacher ranksThe University of Notre Dame Robinson Community Learning Center (RCLC) will expand its licensed preschool program and boost the ranks of local preschool teachers with a grant from Early Learning Indiana.
- Using anti-racist messaging boosts credibility of human rights groups, Notre Dame study showsHow can human rights groups criticize governments' human rights violations without appearing racist or fueling racism toward diaspora groups? New research by a University of Notre Dame human rights expert sheds light on the complex relationship between race and human rights, especially as it plays out between human rights groups and governments.
- Merry Christmas from Notre DameMay we be the seekers of truth, the sustainers of hope, and the builders of bridges that our world needs. – Rev. Robert A. Dowd, C.S.C., University President
- New global tool measures climate resilience at the city levelWhat is the best use of limited resources for cities to adapt to climate change? To help answer this question, governments and organizations now have a critical new resource developed by a team at the University of Notre Dame: the Global Urban Climate Assessment. It is a decision-support tool that offers leaders a way to understand and compare city vulnerabilities, assess adaptation plans and develop resilience.
- AmeriCorps awards Notre Dame funding to connect tutors with evidence-based practicesThe University of Notre Dame has received a $640,108 grant from AmeriCorps, the federal agency for national service and volunteerism, through its Volunteer Generation Fund. The grant will support the capacity-building efforts of Tutor-ND, Notre Dame’s learning design hub that connects tutors with cognitive science and evidence-based tutoring practices.
