PKD Life - Summer 2022 - 19

PHOTOGRAPHY BY NICOLE LOEB
Melissa and
Steven White.
PKD Foundation
Forms ARPKD
Task Force
Aware that research, understanding of, and
treatments for ARPKD lag behind those
for ADPKD, the PKD Foundation has established
a task force to focus specifically on
ARPKD, which affects 1 in 25,000 babies
born every year.
The task force consists of ARPKD physician
scientists, Foundation members, two
ARPKD parents, and one ARPKD patient,
and it convened for the first time in September.
It is focusing both on closing the
education and research gap and on looking
at ways it can help the Foundation directly
address the unmet needs of families facing
this disease. The task force's goal is to
improve outcomes for ARPKD families.
" One of the great things about the task
force is that we are able to learn directly
from parents and from patients about
how they've experienced the disease, from
diagnosis to where they currently are, " says
Chris Rusconi, Ph.D., chief research officer
for the PKD Foundation.
Already, the ARPKD task force is educating
healthcare providers on how to better
support patients. But perhaps most
importantly, it is recommending that the
Foundation accelerate ARPKD research,
including exploring ways to catalyze a
treatment pipeline for ARPKD. To this end,
the Foundation is initiating a project with
the Polycystic Kidney Disease Outcomes
Consortium to create a framework for the
development of treatments for ARPKD. The
consortium engages the Food and Drug
Administration and researchers to help
accelerate therapeutic development. " We
want to start a dialogue with the FDA that
will address how we can develop drugs for
ARPKD, " Rusconi says.
He added that the task force is also interConnect
with social workers: They can " make
things easier for you, " White says. Whether through
the hospital, dialysis center, or doctor's offices,
social workers can find you resources, care, and
information, so take advantage of them.
Assemble the best team of doctors, but question
them, too: " The right team should listen to
your concerns, educate you on the good and bad,
and lay out all the options, " White says. But things
may still fall through the cracks. " Push back if it
feels like something's been overlooked, " she says. If
your gut is telling you something is wrong, trust the
feeling. " Usually, your gut is right, " she adds.
*
TO LEARN MORE ABOUT RESOURCES FOR ARPKD PATIENTS AND FAMILIES, VISIT PKDCURE.ORG/ARPKD
19
ested in making sure that families have
access to clinicians who understand ARPKD
as soon as possible after a child is diagnosed
so that parents get accurate information
based on the specifics of their child's case.
" The Foundation has always supported
ARPKD, but we may not have always clearly
articulated our strategy, " Rusconi adds.
" Our task force is elevating the need for that
strategy ... and giving us a lot more intentionality
about how we are doing this. "
http://www.PKDCURE.ORG/ARPKD
PKD Life - Summer 2022

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