Only 10 percent of rare diseases have an FDA approved treatment. FDA has the power to successfully address the challenges of medicine development for rare diseases, including NPC.
October 2021: We are the patients and parents of patients with an ultra rare and life-threatening disease who are being failed by the systems designed to spur development and expanded access to important treatments. Government, industry and the medical community must come together to protect these children who have a fatal and progressive neurodegenerative disease.
August 2021: In this program, expert clinicians, patients and family speakers shared their perspectives on the effort to find new treatments for NPC, including patient/caregiver preferences, risk tolerance, and opinions about use of the NPC clinical severity scale as a reliable and meaningful tool in clinical research. Our hope was that the FDA would hear our voices and recognize the critical need to be more flexible in reviewing drugs to treat NPC.
August 2021 Listening Session: Letters from the NPC community in advance of, and as follow up to a listening session with FDA, August 2021.
April 2021: In presentations by NNPDF, expert clinicians and patient advocates, the program highlighted many aspects of the severe and devastating impact of Niemann-Pick disease on patients, caregivers and communities. The session also outlines five areas of action that we hope the FDA and the broader research and treatment communities will support moving forward.
March 2019: Video recording of the externally-led, Patient-Focused Drug Development Meeting with FDA in March 2019.
March 2019: Summary report of the externally-led, Patient-Focused Drug Development Meeting with FDA in March 2019.
August 2, 2021
June 17, 2021
June 25, 2021
June 25, 2021
May 24, 2021
April 22, 2021