DENVER (Colo.) and TORONTO (Ont.) – PSC Partners Seeking a Cure is one of five patient-led rare disease groups that, together with collaborative research teams, were selected for support of four-year research projects aimed at advancing understanding of the fundamental biology underlying their rare inflammatory pediatric disease.
Supported by the Chan Zuckerberg Initiative (CZI), the multi-year PSC project will focus on clarifying the cellular mechanisms of pediatric PSC and identifying potential cellular targets for effective therapies. A unique aspect of these projects is that patients will be centered in the work. Each team is co-led by a Coordinating Primary Investigator (PI) and a Patient Organization PI. For the PSC project, entitled Understanding the Cellular Ecosystem of Primary Sclerosing Cholangitis, the Patient Organization PI is Ruth-Anne Pai, PhD, Director of Research Strategy at PSC Partners. Dr. Pai joins a multi–disciplinary and cross-institutional team from Toronto, ON, Canada, made up of Coordinating PI Sonya MacParland, PhD, University Health Network, and PIs Gary Bader, PhD, University of Toronto, and Amanda Riccutio, MD, PhD, The Hospital for Sick Children.
We are extremely grateful to see this important research supported, and we’re excited to work with this team,” says PSC Partners Canada’s President Mary Vyas. “We have been engaged with this research team as a patient partner since 2020. Having immunologist Dr. Ruth-Anne Pai on staff to serve as the patient-PI creates a rich opportunity. It is our hope that delving deeper into the cellular biology of pediatric primary sclerosing cholangitis will lead us closer to our goal of finding treatments and a cure for PSC.”
PSC Partners Founder and CEO Ricky Safer adds, “As a patient living with PSC for many years, I know the value of hope. I applaud CZI for putting a much-needed spotlight on pediatric rare diseases. This research endeavor, with its extraordinary feature of patient-centered collaboration fills me with hope for our children living with PSC.”
About PSC
PSC is a rare liver disease impacting one in every 10,000 people worldwide. The disease does not discriminate based on ethnicity, age, or gender, and the cause is unknown. There are few treatments and no cure. PSC Partners drives research to identify treatments and a cure for PSC while providing education and support for those impacted by this rare disease. Get more information here.
About this CZI Research RFA:
The Chan Zuckerberg Initiative’s (CZI) Patient-Partnered Collaborations for Single-Cell Analysis of Rare Inflammatory Pediatric Disease RFA Grantees brings together patient-led here rare disease organizations and research teams for four-year research projects. Get more information here.
Patient-Led Rare Disease Grantees:
The other rare disease patient organizations selected as CZI grant recipients for this RFA are NEC Society, Children’s Interstitial and Diffuse Lung Disease (chILD) Foundation, RUNX1 Research Program, and Cure JM.
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