We are witnessing a surge in COVID-19 registries for liver diseases, but none are PSC-specific. We believe it will be important to collect information on those of us who have/had/suspect having COVID-19. PSC Partners has thus launched (April 2020) a patient-reported COVID-19 PSC survey. We hope very few of us will have COVID-19, but knowing the impact of COVID-19 on our PSC community may bring a new light on PSC. We will share the aggregated information publicly and only in a de-identified format. Please participate if you have/had/suspect having COVID-19 and you have PSC or had a liver transplant due to PSC. We all know that the complications of COVID-19 can be extremely serious. If you are a caregiver of a PSCer who is unable to complete the survey or is deceased, please complete the survey on their behalf. We are notifying PSC Partners Registry participants of this survey through email, as well as disseminating the survey through social media, and newsletters. We invite PSC patient groups worldwide to join us in sharing the survey. This survey should take no more than 10 minutes to complete. Your privacy is protected because this survey collects no identifying information (for example, we do not collect name, birthdate, or email), and the survey tool does not record your IP address or device identifier. To take the survey, please click here . During these very difficult times, our motto, “Together in the fight, whatever it takes!” is a reminder that none of us in the PSC community are alone. Thank you for joining us! Rachel Gomel PSC Partners Seeking a Cure Registry Director and Registry Coordinator registrycoordinator@pscpartners.org

Little did we know that attending a workshop in Washington D.C. in 2010 would be instrumental in moving PSC Partners towards the unimaginable patient-generated and patient-driven research. In 2010, it was unthinkable that our rare primary sclerosing cholangitis could create a loud enough voice to have a measurable impact on PSC research. In 2009, PSC Partners, in its fifth year of existence, had awarded its first research grants, and we were excitedly searching for more ways of creating PSC awareness. The National Institutes of Health (NIH) workshop, “Advancing Rare Disease Research,” held in Washington D.C., was a pivotal experience for PSC Partners. Hundreds of physicians, academics, patient advocacy groups, drug developers, and regulators from the Food and Drug Administration (FDA) attended this meeting. It was unanimously agreed that having a patient-owned and patient-controlled standardized registry to locate dispersed rare-disease patients was key to increasing engagement in rare-disease research and in facilitating and stimulating research on our neglected diseases. We jumped right in. Starting a registry required knowledge and experience in building and running registries, and, of course, serious funds. PSC Partners Founder and President Ricky Safer and I were volunteers who did not have these important pre-requisites. However, one powerful asset we could count on was our boundless, infinite passion. Shortly after, we were selected to join a registry pilot program run by the NIH Office of Rare Diseases Research (ORDR). We needed medical support. Dr. Christopher Bowlus, Head of Hepatology at the University of California, Davis, and Co-chair of PSC Partners’ Scientific/Medical Advisory Committee (SMAC), never left our side. For a full two years, he attended all meetings with us and became our unfaltering medical advisor and friend. We launched the PSC Partners Patient Registry in 2014. The registry is still in its infancy, and yet has made huge strides. You can explore what's been accomplished to date on the IMPACT page. The 2020 registry upgrade will undoubtedly open many new doors. The registry will no longer be a static picture of a single moment in the PSC journey. This upgrade will allow us to observe changes over time, to add new surveys, and to collect clinical data on each participant. If you are new to this page and to PSC Partners, we encourage you to join our effort to speed up PSC research and to take part in our PSC Partners community by choosing from the myriads of community activities that are wide open to you. Visit the PSC Partners website at www.pscpartners.org . Our registry coordinators are always ready to guide you. You can reach them by email at registrycoordinator@pscpartners.org . We’re together in this fight … whatever it takes! Rachel Gomel Registry Director and Coordinator PSC Partners Patient Registry  Other members of the registry team: Sharon Nanz – Registry Assistant Coordinator Mary P. Vyas – Registry Strategist Dr. Christopher Bowlus – Registry Advisor

If you or a loved one has the rare liver disease primary sclerosing cholangitis (PSC), you are in the right place! This site was developed and is supported by PSC Partners Seeking a Cure, a nonprofit organization dedicated to providing education and support to PSC patients, families, and caregivers. Another vital mission of PSC Partners is to support researchers in their quest to find causes, treatments, and cures for this rare disease. The PSC Partners Patient Registry was initiated in 2014 in response to the difficulty of locating PSC patients and collecting PSC data to conduct research on our rare disease. Our aim with the Registry has been to add the patient’s voice to every facet of PSC research and to facilitate and speed up PSC research towards a cure. All those diagnosed with PSC are encouraged to participate. Parents can join the Registry to enroll their child. A spouse can participate on behalf of their partner. All data in the Patient Registry is de-identified. When a researcher requests information, no identifying information (name, date of birth, address, or phone number) ever leaves the Registry. Researchers, approved by the PSC Partners Patient Registry Team, request de-identified registry data for their research, for patient recruitment in clinical trials, and for developing clinical study protocols. The PSC Partners Patient Registry participants represent an important voice of the PSC community: They engage in research by completing surveys and joining clinical trials. They help us advocate by expressing their unmet needs. When new treatments arrive, the Registry can help track the effectiveness of the new drug and can speed up drug approval. If you or your child appears to be a match for a study or a clinical trial, the registry coordinator will contact you. It would be your choice to connect with the researcher(s) to let them know if you are interested. To join, we request that you complete your profile, the Registry survey, and that you provide a proof of diagnosis which could be a recent or old MRCP or ERCP result, or a doctor’s report that states you have PSC. You can learn more the specifics of the registry by clicking on the various links on this site. We hope you will be inspired to join the PSC Partners Patient Registry. Your voice is crucial for this small and dispersed PSC community! You can help researchers worldwide by taking around 30 minutes of your time to complete your profile -  or your child’s or loved one’s profile. Join the hundreds of men, women, and children who are helping to advance PSC research towards treatments and a cure! We are together in this fight, whatever it takes! Join us! Rachel Gomel, Registry Director and Registry Coordinator Sharon Nanz, Assistant Registry Coordinator Mary Vyas, Registry Strategist If you need assistance, the registry coordinator can assist you at registrycoordinator@pscpartners.org . All rights reserved © PSC Partners Seeking a Cure

PatientCrossroads Launches New Patient Registries. The new rare-disease registries arose from the NIH Global Rare Diseases Registry (GRDR) pilot program. SAN MATEO, CALIF. (PRWEB) SEPTEMBER 04, 2014 PatientCrossroads announces the launch of five new patient registries on behalf of leading rare disease advocacy organizations. The new registries are sponsored by organizations originally selected by the NIH to participate in its Global Rare Diseases Registry and Repository (GRDR) pilot program. At the conclusion of the pilot, a number of organizations chose to pursue the continued development of their own registries and manage them through PatientCrossroads. Five New Patient Registries Launch Through the PatientCrossroads Program: The Barth Syndrome Foundation, Foundation for Sarcoidosis Research, Lymphangiomatosis and Gorham’s Disease Alliance, NephCure Kidney Network and PSC (Primary Sclerosing Cholangitis) Partners Seeking a Cure registries have been launched using the PatientCrossroads registry platform. “We see tremendous value in starting a patient registry as a powerful and practical step toward advancing the research initiatives of the Sarcoidosis Research community. The registry has been collecting data since June and we have registered over 800 patients in that short time.” said Ginger Spitzer, Executive Director of the Foundation for Sarcoidosis Research. “The PatientCrossroads approach fits well with the foundation’s goal to have promising therapies move quickly from the laboratory to the patient.” The registries will be able to collect patient-provided data in a manner that contributes to pan-disease research. “One of the important learnings from the GRDR pilot was that patient registries are expensive, complex and challenging for rare-disease organizations to maintain on their own,” said Kyle Brown, founder of PatientCrossroads. “Only by using a common platform can the complexity and cost of managing patient registries be minimized to make them available to any rare disease community. By using our platform, disease organizations get the full benefits of patient registries while also freeing more of their capital for other purposes, such as funding research programs and directly supporting patients.” About PatientCrossroads: Since 2005, PatientCrossroads has pioneered the use of patient registries to accelerate therapeutic development and empower patients. PatientCrossroads registry programs connect patients with researchers, advocates and industry organizations working to understand or treat specific diseases and conditions. Dedicated to openly accessible registry programs, PatientCrossroads has been recognized for its efficacy through national and international awards and partnerships. Its groundbreaking CONNECT patient registry platform allows de-identified patient information from multiple diseases to be shared and used to research diseases, find new treatments, and better educate and support patients and their families. For more information, visit http://www.patientcrossroads.com .   https://www.prweb.com/releases/2014/08/prweb12133961.htm

Survey Results: Oral Vancomycin and Insurance Coverage PSC Partners received a request from patients to survey the PSC patient community on their experience with insurance coverage of their medication, particularly oral vancomycin, in advance of Rare Disease Day February 28, 2023. The data was used in part to inform legislative advocacy in support of HR6160, Access to Rare Indications Act of 2021. A short, IRB-approved survey was administered through the PSC Partners Patient Registry. The survey was open for 5 days in February 2023 (February 22 – February 26). The survey is now closed. Survey Results can be downloaded as a pdf below. Thank you! Oral Vancomycin and Insurance Coverage Survey Results v3

News from the EASLCongress 2023 meeting in Vienna From the "late-breaking" abstract session on June 24, 2023, results of the pre-planned futility analysis of the Phase3 PRIMIS study (cilofexor in PSC) were presented.