We’ve made a difference, but there is more to do, including:
• Translate the research to the patient. Medical research often falls short of its ultimate goal: helping patients. Research should translate from the bench to the bedside, thus improving patient outcomes. This is pinnacle for those we serve.
• Fund disease specific research, which is only limited by family support. As a patient advocacy organization we have the ability to improve patient outcomes and what we learn about ARPKD/CHF can help more common diseases.
• Determine clinical outcome measurements for both the kidney and the liver, so that when a drug for ARPKD and/or CHF is ready to move into clinical drug trials it is not delayed due to an inability to measure a drug’s effectiveness.
• Generate and foster future professional collaborations for research direction and priorities, partner with others, and share resources.
• Maintain a secure registry of data for future clinical trials and research with highest protections that can communicate safely with others.
• Promote ARPKD/CHF further within professional networks and circles.
• Position the patient voice, perspectives and questions on committees, thus creating a stronger influence for our disease.
• Create and support legislation to increase awareness and funding.
• Develop transplant resources specific to our disease and ARPKD/CHF Healthy Transitions – from Pediatric to Adult Healthcare.
Future Organizational Goals:
• Build infrastructure.
• Cultivate future patient advocates and leaders.
• Increase awareness and generate support for our mission, which includes treatments and medical care and most importantly patient outcomes.