In 2001 research and awareness were minimal and almost universally ARPKD was considered a hopeless condition. More often than not parents were given antiquated, obsolete information with little to no hope for survival. This happens even today, but to a much lesser degree. Professionals are better educated and enhanced general medical care has improved outcomes. Infants once considered hopeless are more often provided medical care and survival into adulthood is not uncommon. Some of our accomplishments include:
- Created “a voice” for this disease, where once there was none.
- Partner with the world’s leading funding agency, the National Institutes of Health, for “Clinical Investigations into ARPKD and CHF”
- Collaborate with the National Disease Research Interchange to connect patients with researchers.
- Developed and provide free educational materials and resources to members.
- Generated participation for ARPKD/CHF in the Global Rare Diseases Patient Registry and Data Repository (GRDR).
- Developed Bereavement and Regional Support.
- Created Clinical Care Guidelines and Fact Sheets.
- Produced Public Service Announcements, ie. “Faces of ARPKD/CHF.”
- Co-sponsored the First ARPKD/CHF Medical Symposium.
- Hosted first ARPKD/CHF Conference.
- Created a Research Grant Program, Professional Education, and a Youth Ambassador Project specific for ARPKD/CHF.
- Developed and maintain a secure patient database for future clinical trials with the highest patient protections.
We’ve made a difference, but there is more to do.