Explore patient and physician resources, consider advancing research by participating in a clinical study, learn more about the PKD Foundation’s investment in ARPKD research, and mark your calendar for national awareness events.
ARPKD Patient Handbook
Watch ARPKD sessions from this year’s virtual PKDCON
ARPKD Clinical Studies
ARPKD and ARPKD-Related Diseases Database
The University of Alabama at Birmingham Hepato/Renal Fibrocystic Disease Core Center (UAB HRFDCC) has developed a unique set of clinical, genetic, and educational resources for ARPKD and other recessive forms of renal cystic disease.
If your child has PKD, consider sharing your experience and helping advance research. Children’s National researchers have partnered with other PKD researchers globally to better understand pediatric PKD. No travel to a clinic is needed to participate.
Novel Imaging in ARPKD
Novel MRI Techniques to Evaluate ARPKD Kidney and Liver Disease Progression
Receive notifications when there are clinical studies in your area.
Investment in ARPKD Research
PKDF has invested over $2 million since 2006 for research, support, education and awareness for ARPKD, ADPKD in children, and congenital hepatic fibrosis (CHF), a disease closely associated with ARPKD. The Foundation also strives to provide support through funding and collaborations in the following ways:
Dedicated ARPKD research funding
Over the past five years, PKDF has awarded biennial research grants (transitioned to annual as of 2019) to outstanding PKD researchers to increase understanding of the genetic and pathological processes involved in PKD.
Although the results of all PKDF-funded scientific projects may ultimately benefit and contribute to discoveries in PKD in children, you can read more about projects related to ARPKD here.
2022 Grants Supporting ARPKD research
- Katherine Dell, M.D. is a pediatric nephrologist at the Cleveland Clinic Children’s. Her project is focused on novel MRI fingerprinting of congenital hepatic fibrosis in ARPKD.
- Charles DeRossi, Ph.D. is an Assistant Professor in the Department of Pediatrics at the Icahn School of Medicine at Mount Sinai. His project is focused on glycosylation as a regulator of liver disease in ARPKD.
2021 Grants Supporting ARPKD research
- Erum Aftab Hartung, M.D., MTR, is a pediatric nephrologist at the Children’s Hospital of Philadelphia (CHOP). Her project is focused on intracranial aneurysms and vascular abnormalities in ARPKD.
- Professor Melissa Little, BSc PhD GAICD, FAAHMS, FAAS is the Theme Director of Cell Biology at the Murdoch Children’s Research Institute in Melbourne, Australia. Her project is focused on in vitro modelling of ARPKD.
Donate ARPKD affected kidneys and livers
Our PKD tissue donation program helps patients contribute to the advancement of our understanding of PKD.
Representation in PKDF Scientific Advisory Committee
Made up of 14 prestigious PKD physicians and scientists, the Scientific Advisory Panel (SAP) oversees our research and medical programs aimed at discovering and delivering treatments for PKD. The SAP meets throughout the year to discuss relevant medical issues, provide guidance to our staff, and review and approve research applications for grants and fellowships in the field of PKD science. All our materials and publications are approved by SAP members, who possess the highest level of experience and knowledge in PKD clinical and scientific work.
Two current members in particular are champions in the pediatric PKD space:
Erum Aftab Hartung, MD, MTR, is a pediatric nephrologist at the Children’s Hospital of Philadelphia (CHOP). Her research interests include ARPKD, development of imaging biomarkers of kidney and liver disease, and neurocognitive outcomes in children with chronic kidney disease. She currently serves as chair of the Research Committee of the American Society of Pediatric Nephrology, and is the Associate Program Director for the pediatric nephrology fellowship at CHOP. Dr. Hartung’s research is funded by grants from the National Institute of Diabetes and Digestive and Kidney Diseases (National Institutes of Health) and the University of Pennsylvania. Dr. Hartung is on the faculty of the Perelman School of Medicine at Penn as Assistant Professor of Pediatrics. She lives in Swarthmore, PA with her husband and two children.
Max C. Liebau, MD, is a clinical consultant pediatric nephrologist at the Department of Pediatrics at the University Hospital Cologne, Germany, where he holds positions as Head of the Social Pediatric Center for Chronically Ill Children and Head of Translational Pediatric Nephrology. Dr Liebau combines his clinical training as a pediatric nephrologist with his experience in cellular and molecular biology obtained in the Nephrology Research Laboratories in Freiburg and Cologne, Germany and at the University of California, Santa Barbara. His group follows a translational research approach to study genetic kidney diseases with a special focus on Autosomal Recessive Polycystic Kidney Disease (ARPKD). The group aims to understand the molecular function of the ARPKD protein fibrocystin and to characterize clinical long-term courses of ARPKD as a basis for the identification of clinical and/or biochemical risk markers of disease progression. Dr Liebau initiated and is currently leading the international ARPKD registry study ARegPKD and is a co-initiator of the pediatric ADPKD registry study ADPedKD. His research is funded by the German Research Council and the German Federal Ministry for Education and Research amongst others.
ARPKD Task Force
The ARPKD Task Force was formed by the PKD Foundation Board of Directors and includes ARPKD physician scientists, parents of children with ARPKD, and executive staff members. Established to guide the Foundation’s efforts to address the important and unmet needs of the ARPKD community, the ARPKD Task Force will design a comprehensive strategy including research programming, a communication plan, and an advancement campaign with the goal of improving outcomes for ARPKD families.
Supported Scientific Meetings
FASEB – PKD: Hurdles and Advances in Molecular Mechanisms and Therapies
June 24-29, 2022 in Lisbon, Portugal
2021 PKD Regulatory Summit
May 19–20, 2021 (virtual)
View session recordings here.
Read the resulting publication:
Perspectives on Drug Development in Autosomal Recessive Polycystic Kidney Disease
May 3–5, 2019 in Chicago, IL
April 21–23, 2017 in New York City
April 5–7, 2013 in New York City
June 29 – July 1, 2018 in Kansas City, MO
June 25–30, 2017 in Big Sky, MT
April 30 – May 3, 2016 in Baltimore, MD
2022 Awareness Events
Follow the PKD Foundation on Facebook, Twitter, Instagram, and LinkedIn for event updates and download and share our PKD in Children handout.
14 | National Donor Day
28 | Rare Disease Day
National Kidney Month
10 | World Kidney Day
National Donate Life Month
22 | Blue & Green Day
24 – 30 | National Pediatric Transplant Week
4 | PKD Awareness Day
National Minority Donor Awareness Month
15 | National Pregnancy & Infant Loss Remembrance Day
National Family Caregivers Month
Page last updated August 2022