PSC Partners Seeking a Cure Surpasses 1,000 Patients in International Registry and Discovers Unexpected Misdiagnoses

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Enrolling Patients with Rare Liver Disease in International Registry Speeds Up Research

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PSC Partners Seeking a Cure and PSC Support, a nonprofit PSC patient organization based in the UK, have been working on an educational campaign to promote a better understanding and more accurate diagnosis of these two rare liver diseases.

PSC Partners Seeking a Cure, a nonprofit organization that provides education, support, and research funding for the rare liver disease primary sclerosing cholangitis (PSC), has recruited more than 1,000 patients for their international patient registry.

The international PSC Partners Patient Registry was established in 2014 in collaboration with the NIH Office of Rare Disease Research and was created to bring information on PSC and those affected by the disease into a single place. Because PSC is rare, it is difficult to conduct large clinical trials and develop treatments for the disease. An international registry enables academic medical centers and pharmaceutical companies to easily locate patients who opt in to the patient-driven registry.

The registry collects data including information on diagnosis, family history, quality of life, medications, surgeries, overlapping diseases, and patient demographics. With this information, researchers can find appropriate patients for clinical trials, facilitate drug safety monitoring for new drugs, and allow for patient participation at each level of research.

“Our registry is designed to be a resource for researchers who are working to develop much-needed therapies for PSC, since the disease currently has no effective treatments. We are thrilled to have reached such a large milestone, and hope that bringing together more than 1,000 PSC patients will speed up research by enabling easier recruitment of participants for clinical trials,” said Ricky Safer, CEO and founder of PSC Partners Seeking a Cure.

An unexpected result of enrolling more than 1,000 patients in the registry is that some of those who tried to enroll learned -- after answering a series of questions about their diagnosis and symptoms -- that they do not actually have PSC. Instead, they have primary biliary cholangitis (PBC), a distinctly different disease that also affects the bile ducts.

This misdiagnosis is troubling, given that effective treatment exists for PBC and patients who thought they had PSC may have been missing out on treatment that would slow the progression of PBC.

PSC Partners Seeking a Cure and PSC Support, a nonprofit PSC patient organization based in the UK, have been working on an educational campaign to promote a better understanding and more accurate diagnosis of these two rare liver diseases. Given the similarity of their names, PSC and PBC have been confused in medical publications, among regulatory bodies, and in medical practice -- as well as in individual patient diagnoses, as seen in the PSC patient registry.

Without an accurate diagnosis, patients with PBC would be missing out on important treatment, and PSC patients, who (unlike PBC patients) are at an increased risk for colon and bile duct cancers, would not get critical cancer surveillance.

"We are deeply committed to raising awareness about PSC, and that includes ensuring that the important differences between PSC and PBC are recognized. Patients should have appropriate diagnosis and care, and importantly, access to the right clinical trials," said Martine Walmsley, chair of PSC Support UK.

To learn more about the PSC Partners Patient Registry, or to enroll, visit

About PSC Partners Seeking a Cure
PSC Partners Seeking a Cure is a 501(c)(3) nonprofit organization that provides education and support for people affected by the rare liver disease primary sclerosing cholangitis (PSC). Formed in 2005, the organization raises funds to research the origins, treatments, and a cure for the disease. Education and research initiatives of PSC Partners Seeking a Cure include annual conferences for PSC patients and their caregivers; a competitive PSC research grant program; an IRB approved patient-driven registry created in collaboration with the NIH and overseen by a medical committee including members of PSC Partners; a comprehensive website for patients, caregivers, and medical professionals; local and online support groups; and a PSC literature site, the most extensive online collection of studies on PSC and related diseases. To learn more, visit

About PSC Support
UK based PSC Support is a nonprofit charity organization whose mission is to improve the lives of people with primary sclerosing cholangitis. PSC Support informs, funds and supports high quality scientific research, provides emotional support and networking to PSC patients and their families in over 150 countries, takes action to improve PSC related liver disease and organ transplant policies and services, and provides education and awareness about PSC to patient communities and medical professionals. To learn more, visit

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Carrie Yamond
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