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.
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.
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.
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