Two and a half decades. One community.
The story of the ARPKD/CHF Alliance is the story of families refusing to let their children be forgotten by research and policy.
- 2001
Founded by Families
The ARPKD/CHF Alliance is founded by parents of affected children seeking community, research, and answers.
- 2002
First Online Resources, Registry & Gathering
Established the first accurate internet information — including prenatal, diagnosis, and bereavement resources — and a patient registry with Institutional Review Board protections. Presented the ARPKD/CHF database at the NIH PKD Strategic Planning Meeting, requested that ARPKD/CHF be added to the ADPKD CRISP studies, and held the first social gathering of ARPKD/CHF families at Spring Gulch Camp Resort in Pennsylvania.
- 2003
NIH Natural History Study Launched
Generated the largest research study in the world specific to ARPKD/CHF — “Clinical Investigations into Autosomal-Recessive Polycystic Kidney Disease and Congenital Hepatic Fibrosis” — at the NIH in Bethesda, Maryland. This was the first time the disease was comprehensively and prospectively evaluated.
- 2004
Added to NIH's Liver Action Plan
Petitioned for Congenital Hepatic Fibrosis to be added to the NIH Liver Action Plan, helping shape future research direction.
- 2005
First ARPKD/CHF Medical Symposium & CHF Research Grant
Co-sponsored the first ARPKD/CHF Medical Symposium, in which clinicians and researchers determined that “liver disease is significant in ARPKD” — leading the Alliance to fund a $50,000 research grant for Congenital Hepatic Fibrosis.
- 2006
Clinical Care Considerations & National Symposium Report
Created Access to Care documents and Clinical Care Considerations for informed, individualized decision-making, and developed the Professional Educational Project (PEP) to replace antiquated information that often left families with little or no hope. The Alliance also helped sponsor a 1.5-day NIH symposium (NIH Office of Rare Diseases and NHGRI) on the present status and future of ARPKD/CHF research — resulting in a published summary statement that changed the direction of the field.
Read the NIH / Office of Rare Diseases summary statement → - 2007
New Website & Regional Support
Built an extensive new website as a go-to place for information and help, and developed regional support.
- 2008
First Family Conference
Held the first family conference specific to the disease.
- 2009
Research Breakthrough
The fibroblast cell responsible for CHF is identified through research supported by the ARPKD/CHF Alliance.
- 2010
Research Papers Published
Research papers published from the Natural History Study established for the first time that kidney size does not correlate with kidney function in ARPKD — the opposite of ADPKD, as determined by the ADPKD CRISP studies.
- 2011
Supported PKD Research Meeting
Supported a PKD meeting hosted by the Federation of American Societies for Experimental Biology (FASEB).
- 2012
NDRI Partnership & Family Gathering
Partnered with the National Disease Research Interchange (NDRI) to support research, and participated in the Midwest Family Gathering Family Fun Day in Kenosha, Wisconsin.
- 2013
Patient Registry & Policy Advocacy
Created the ARPKD/CHF Alliance Patient Registry within the NIH Global Rare Diseases Patient Registry and Data Repository (GRDR) and Office of Rare Diseases Research (ORDR); advocated for policy changes at the United Network for Organ Sharing (UNOS) for CHF organ allocation; and generated interest for a Consensus Medical Management meeting for ARPKD/CHF.
- 2014
Quality of Life Research & Advocacy
Represented ARPKD/CHF at the Drug Information Association’s 50th Annual Meeting — one of the largest pharmaceutical meetings in the world — participated in the NIH’s NCATS Global Rare Diseases Patient Registry Data Repository, and created the first ARPKD/CHF Quality of Life research.
- 2015
Advocacy Training & West Coast Gathering
Coordinated Advocacy Training to cultivate future patient advocates and leaders, and held A Friends and Family West Coast ARPKD/CHF Gathering.
About the West Coast Gathering → - 2016
A Transformed Outlook
With continued drive — and advancements in prenatal sonogram technology, neonatal care, and genetic medicine — the outlook for children diagnosed has been completely transformed. The disease is no longer considered uniformly fatal, and the vast majority of those affected now survive well into adulthood.
- 2018
Family Conference & First Board Meeting
Held a family conference; held the first face-to-face Board Meeting; and represented ARPKD/CHF at NORD’s 2018 Rare Diseases and Orphan Products Breakthrough Summit.
- 2019
PCORI Ambassador Program
Participated in the PCORI Ambassador Program and represented ARPKD/CHF at its Annual Meeting.
- 2020
Virtual Conference & PCORI
Held a virtual ARPKD/CHF conference connecting families and clinicians, and represented ARPKD/CHF at PCORI’s Annual Conference.
- 2021
Care About Rare Campaign
The Care About Rare virtual Walk/Run/Bike campaign launches as a community-wide event.
- 2022
RARE Health Equity Summit
Attended the RARE Health Equity Summit to represent ARPKD/CHF.
- 2023
PCORI Annual Meeting
Represented ARPKD/CHF at the PCORI Annual Meeting.
- 2024
RARE Drug Development Symposium
Participated in the RARE Drug Development Symposium.
- 2026
Two and a Half Decades, One Community
Twenty-five years of family support, advocacy, and research progress — from 2001 to 2026.