Externally Led Patient-Focused Drug Development (EL-PFDD) Meeting on Fabry Disease
- You'll be able to submit comments on your experience with Fabry disease before the meeting
- The link to join the meeting will be available on the day of the meeting (September 19th)
Take the patient survey
Who benefits from EL-PFDD meetings?
Food and Drug Administration (FDA)
Patient advocacy groups
Hear from Previous Patient and FDA Participants
I can say with certainty…that the stories that you shared with us today, your experiences with the disease will certainly impact how we at FDA will think about this disease, will approach our discussions with sponsors moving forward, and will really shape the development in the future of therapies for this disease.
Being able to tell my story, be heard, and people empathizing and understanding the struggles this disease faces you with is indescribable.
I think that your stories really highlight the urgency with which we need to find effective treatments for this disease, and also treatments that have better safety profiles than those that are currently out there.
I liked that everyone was invited to speak freely and participate. It was an excellent opportunity to tell our stories.
If I take the Patient Survey, what will you do with the information I provide?
Is there a charge to attend the meeting?
How do I register to attend the EL-PFDD meeting?
When and for how long is the EL-PFDD Meeting on Fabry disease?
Is this EL-PFDD Meeting on Fabry disease for pediatric patients?
What happens after the meeting?
The National Kidney Foundation (NKF) is the oldest and largest non-profit health organization dedicated to preventing kidney diseases and improving the health and well-being of individuals and families affected by kidney disease. Education of patients and healthcare providers is a big part of the NKF’s work, and we support kidney patients by providing all sorts of educational materials, programs, and patient services.
The mission of the Fabry Support & Information Group (FSIG) is to raise awareness of Fabry disease and its symptoms, to advocate for community needs and to lead the fight for the most effective treatment possible and eventually a cure. FSIG provides a link for the Fabry community to information, each other, the medical community, researchers, the pharmaceutical industry, and regulatory authorities. FSIG strives to support affected families and individuals lead fuller lives.