Dear Registry Participants (Current and Future),  This year is the 10th anniversary  of the PSC Partners Patient Registry, a perfect time for renewal, for the upgrade you can experience when you log into the Registry. What you will experience is   only the tip of the iceberg . The Registry is fulfilling a PSC Partners dream transformed into the tangible goal of facilitating and speeding up research towards PSC treatments and a cure.  This progress could only take place thanks to all of you participating in the Registry. With your enthusiasm to embark on this journey, you’ve helped open many new paths leading to patient-centered research, advocacy for our unmet needs, and empowerment of our 2,500 voices. Thanks to all 2,500 Registry participants, the Registry is thriving.  A New Registry Experience I will start with the tip of the iceberg, what you will experience when you enter your new space in the Registry and its website. (Please check out the Registry website  for the short video tutorials created to help you log in and navigate through the new features.) The platform has multi-language capabilities for our international participants or for those whose native language is not English. (The Registry platform supports English, Spanish, French, Italian, and German. The surveys are in English at this time but will be translated into these languages in the near future.)  You can now securely share your experience with your physicians and family members. You will be able to upload medical documents, MRIs, school forms, genetic reports, and more. You can track your symptoms and activities and share them with your physicians. The Registry has the capability to keep track of your adherence to medications and adverse events. You can create journal entries to describe the care you are receiving or the symptoms you are experiencing. The Registry website  is regularly updated with news on clinical trials, physicians treating Registry participants (Physician Map), the impact you are having on PSC research, and general news. To access your new Registry home, go to www.pscpartnersregistry.org  and select “Join Now” if you are NOT an existing participant. However, if you are an existing Registry participant, Select “Log In'' from our home page at pscpartnersregistry.org , enter your email address, and click on “Forgot your password?” in order to create a new password. (You must create a new password, even if you remember your previous password.) There are video and written tutorials  on the website to help you. Your Collective Impact Matters The above features are what you can  see. Below are a few of the PSC Partners initiatives that lie beneath the visible tip of the iceberg. They were each enabled by your participation in the Registry.   Your de-identified responses to Registry questions have helped shape the PSC Partners Strategic Research Plan . Over 800 people with PSC responded to Our Voices Survey  and expressed their priorities, which PSC Partners scientists and PSC thought leaders translated and transformed into a research plan. An in-person inaugural meeting brought together the PSC Partners International Collaborative Research Network ( ICRNetwork ) in 2023. Patients, clinicians, researchers, data analysts, and regulators (FDA representatives) formed working groups to discuss your research priorities and ways of implementing them with scientific rigor. Projects are already ongoing. WIND-PSC  was created in response to your research priorities and unmet needs. You let us know that your top research priority was to slow down disease progression.  Gaps were identified, one of which is the absence of dependable and solid PSC biomarkers, which are a requirement to determine whether a PSC drug is effective and can be approved. Over 10 medical centers and 2,000 people with PSC will be participating in this massive initiative. We will ask you for your participation.    Registry data informed us about the PSC symptoms that affect your quality of life, and, in response, a Symptom Assessment Program  (SAP) was created. Two experts interviewed some of you to listen to your description of your PSC symptoms. They are currently translating what they heard from you into validated tools that can be used in clinical trials to objectively measure changes in your symptoms. It is only through your participation and engagement that these tools can be developed and be used to determine the success of a new PSC drug.   Joining a rare disease registry is a powerful act that goes beyond simply providing data. It’s about building community, advocating for change, and actively participating in research. By adding your voices to the Registry, you are powerful partners in the quest for a cure. Patients are moving mountains, and the decision-makers are clearly hearing us. Please know that each email we send you is tied to our collective mission. Please do your very best to respond if you possibly can.  The Registry Team sending you these messages is comprised of three Moms whose children have PSC and two people with close connections to PSC. All of us together are passionately working towards treatments and a cure.  JOIN THE RACE! YOUR JOURNEY MATTERS. Together we are creating a world where a PSC diagnosis comes with a cure! Rachel Gomel  Director, PSC Partners Patient Registry The 3 Unstoppable PSC Moms who Believe in the PSC Community’s Unshakable Power! THE PSC PARTNERS REGISTRY TEAM Rachel Gomel , Director Sharon Nanz , Registry Curator Mary Vyas , Registry Strategist Susan O’Dell , Registry Analyst Brian Thorsen , Registry Webmaster We’re together in the fight, whatever it takes!

On February 4, 2026, the initial study details for Ipsen’s Phase 3 ELASCOPE clinical trial for elafibranor for treatment of PSC  were listed on ClinicalTrials.gov . Study enrollment is anticipated to start around April 2026 and key details of the study design are now available. “PSC Partners is thrilled to see Ipsen move forward with this global Phase III study,” said Ricky Safer, Founder and CEO of PSC Partners. “This is a key step forward in fulfilling our mission of finding a cure for PSC. We are grateful to Ipsen for their commitment to those affected by PSC and to all patients whose prior participation in research have made this milestone study possible.”   “Ipsen is honored to have launched a Phase III clinical study for people living with PSC,” added Jennifer Schranz, Senior Vice President, Global Head of Rare Diseases at Ipsen. “We are deeply committed to advancing potential treatments for rare liver diseases where there is a significant unmet need and few options for patients currently exist.”   Below is a summary of elafibranor and the pivotal global Phase III study: About Elafibranor: Elafibranor is a PPARα/δ agonist taken as an oral pill which can potentially treat PSC by decreasing bile acid synthesis and toxicity, increasing bile acid secretion, and decreasing inflammation. Together, these effects may lessen damage caused by cholestasis in PSC. The Phase 2 study  of elafibranor in PSC (the ELMWOOD trial) showed dose-dependent reductions in ALP and other markers of liver injury and fibrosis, as well as a favorable safety profile. Elafibranor is already approved by regulatory agencies for treatment of primary biliary cholangitis (PBC) as a second-line treatment when UDCA (Urso) is not effective for a patient. Study Details: The ELASCOPE trial anticipates enrolling 350 patients across approximately 200 sites globally. The study will be open to adults with large duct PSC and no history of liver transplant or decompensated cirrhosis (complications associated with advanced disease). Patients will be randomly assigned to receive elafibranor or a placebo.   Concurrent UDCA usage is allowed, along with most other stable ongoing treatments. Medications that are NOT concurrently allowed include, but are not limited to: norucholic acid, fibrates, seladelpar, and glitazones.   Study participation is expected to last up to five years to collect sufficient outcomes data (events associated with disease progression) to allow for effective comparison of the group receiving elafibranor to the placebo/control group.   For more information, including inclusion and exclusion criteria, please visit the study listing on ClinicalTrials.gov . PSC Partners is encouraged by the news of elafibranor advancing to Phase 3, the critical study phase to determine whether a drug will receive market approval by the FDA and other regulatory agencies. We will continue to share more information about this study as it becomes available.

Dear Registry Community, 2025 has been a banner year for the Patient Registry. Between important new surveys, Registry-led publications, external research support, and the launch of Spanish and French translations, the Registry is reaching ever greater heights. At the heart of the Registry’s success, making everything we do possible, is your active participation in the Registry. With 223 new participants in 2025, the Registry now represents over 2,770 people living with PSC from 55 countries. And with 2,083 surveys completed last year alone (more than double the previous year!), your voice is heard loud and clear. Your contributions to research are invaluable, and we will ensure that all the information you have shared will tangibly drive research forward. Looking ahead to 2026, we aim to further expand the Registry’s global reach and to bring the insights made possible by your data into new publications, which will be shared back with the community. You can also look forward to a new survey launching later this year to capture transplant and post-transplant experiences. On behalf of the Registry team, we wish you all good health and good care in 2026. May this year continue to bring us ever closer to a cure for PSC. - Brian Thorsen, Director, PSC Partners Patient Registry Meet our New Assistant Director Assistant Registry Director Fernanda Quevedo  joined the Patient Registry Team in early January, but she is not new to PSC research. Her work experience includes several years as the research program/project manager at the Resnek Family Center for PSC Research at Brigham and Women’s Hospital in Boston, Massachusetts, where she worked with Dr. Joshua Korzenik and his team. Now Available: Clinical Survey 2026 The new Clinical Survey 2026 was just released and is available in your Registry account. Have you ever wondered why the Registry updates this survey every year and why we request that you update it annually? New Language Support: Spanish and French We are excited to announce that key parts of the Registry’s internal data platform, including most current surveys, are now available in Spanish and French . In addition, the public website also includes newly translated sections. With these translations, the Registry improves its global access and representation for those with PSC. Cholangiocarcinoma (CCA) Survey Results In Spring 2025, the Registry conducted the PSC and Cholangiocarcinoma (CCA) Patient Survey. This survey collected information on current practices of monitoring for CCA (also known as bile duct cancer), educational resources used by patients, and communication between patients and providers regarding CCA. Results from the survey were presented at AASLD’s The Liver Meeting 2025. Stay Connected with PSC Partners Sign up to receive the information that matters most to you— from research and volunteer openings to our three e-newsletters:  The Beacon - new, scientific research newsletter providing a comprehensive look at how patient-driven research and donor contributions make an impact. The Duct - the latest PSC news, updates and events The Viaduct - Patient Registry news For your privacy, any connection to the Registry will not be recorded in this signup form.

Help researchers worldwide unlock the mysteries of primary sclerosing cholangitis (PSC). Complete your profile and join the PSC Partners Patient Registry to contribute your voice and experiences to our collective knowledge of PSC! You are an important piece of the PSC puzzle! Join the PSC Partners Registry to take action and drive PSC research forward. JOIN NOW LOG IN The Registry is a platform to amplify your voice in PSC research. The PSC Partners Patient Registry is a digital platform where basic data on people living with primary sclerosing cholangitis (PSC) is collected and stored. Data is primarily collected through online surveys completed by the participant living with PSC, or a qualified caregiver. Join the Registry to... Complete surveys that confidentially capture your experiences and address key questions in PSC research; Document and track your medical history with PSC, including treatments, symptoms, and healthcare providers; Elect to receive information about participating in research and clinical trials . You will be notified if you qualify for a study and can choose to contact the study team. The PSC Partners Patient Registry is open to everyone with PSC , wherever you are in the world. Parents and caregivers can join on behalf of their children. Any information you share is de-identified before sharing with researchers. No patient-identifying data ever leaves the Registry. LEARN MORE LATEST NEWS Elafibranor Advances to Phase 3 Clinical Trial for Treatment of PSC The Viaduct - Issue 10, Winter 2026 Patient Registry Now Accessible in Spanish & French Now Available: Clinical Survey 2026 What is the PSC Partners Patient Registry? What can I expect after I join? Learn More Join the Registry to add your information to our collective voice. Join Now Interactive map of healthcare providers provided by PSC Partners Patient Registry participants. Find PSC Specialists Data is power Thanks to people like you, the experience of living with PSC can be understood like never before. The more health information we collect through this patient registry, the closer we can get to helping our research community find treatments that work. Check out this video to learn more. "Where well-implemented registries and active patient organizations exist, the likelihood for developing a treatment for the disease in question is increased." (Eurordis-NORD-CORD) For questions, email registrycoordinator@pscpartners.org Visit PSC Partners website: pscpartners.org Join the Registry today! We're together in the fight, whatever it takes. With appreciation to Espen Bunæs for design support

Help researchers worldwide unlock the mysteries of primary sclerosing cholangitis (PSC). Complete your profile and join PSC Partners Seeking a Cure in advancing PSC research towards a cure. Your participation is important. Registry Overview WHAT IS THE REGISTRY? PRIVACY AND SECURITY CURRENT SURVEYS OTHER REGISTRY FEATURES The PSC Partners Patient Registry is a digital platform where basic data on people living with PSC is collected and stored. Data is primarily collected through online surveys completed by the participant living with PSC, or a qualified caregiver. Confidentiality and privacy measures are built into the Registry to ensure participant privacy. This data is then used to accelerate PSC research and advocate for the unmet needs of PSC patients. Researchers request de-identified patient data which is analyzed to gain new insights on PSC. Click here to view the current list of publications made possible through the Registry. Researchers can also request the Registry's assistance in recruiting for a clinical trial or study. The Registry team identifies participants who best meet the study's inclusion and exclusion criteria and privately send these participants information about the study. The participants can then choose to contact the study team if they want to learn more and/or are interested in joining the study. This practice ensures that the participant's identity is withheld from researchers and that it is solely the participant’s choice to join a study. The Registry also provides current news on clinical trials and studies in a few additional ways: the Ongoing Clinical Trials page, the Publications Related to Clinical Trials page, the VIADUCT Newsletter , and News posts . This information is publicly available under the "NEWS AND RESOURCES" and the “CLINICAL TRIALS” sections on the navigation menu. No identifying data ever leaves the Registry. The Registry’s operating software, Matrix, was designed and is operated in accordance with applicable US privacy protection provisions of HIPAA,¹ the European GDPR,² and FDA regulations.³ These regulations require specific documented operational controls and security procedures. Information that you provide to the Registry via the Matrix website is encrypted* and stored on secure servers with restricted physical access. To further protect the best interests of Registry participants, the Registry is annually reviewed by an Institutional Review Board (IRB) and all Registry team members have current HIPAA training. Any personal information that could be used to identify you or your family is labeled as Personally Identifiable Information (PII). Your PII is securely stored and encrypted. Only authorized Registry staff can access your PII and contact you if needed. Information that has had all PII changed to a code is called "de-identified." Only de-identified data is ever shared with researchers. ¹HIPAA: Health Insurance Portability and Accountability Act of 1996 ²GDPR: General Data Protection Regulation (GDPR) ³FDA regulations: Food and Drug Administration Title 21 of the Code of Federal Regulations Part 11 (21 CFR part 11) *Encryption means that even if a third party were to obtain access to the data, they must have access to a long “passkey” to decrypt and view the original information. Without having the passkey, it takes decades for even the most powerful supercomputers to figure out the original passkey. Clinical Survey The Clinical Survey is the heart of the Registry. This survey collects information on a wide range of topics, such as PSC diagnosis, symptoms and complications, other diseases, family history, and more. A new Clinical Survey is available each year so that the Registry can track changes over time. Health Equity Survey The Center for Disease Control defines health equity as the state in which everyone has a fair and just opportunity to attain their highest level of health. This survey seeks to build an understanding of the impact of PSC and barriers to receiving high quality care. PSC Flare or Cholangitis Attack Patient Survey Many people living with PSC are intimately familiar with what a cholangitis attack feels like. However, there is not yet a widely agreed-upon medical definition for this major complication associated with PSC. This survey aims to capture participants' history of cholangitis and symptoms during their most recent attack. Healthcare Provider Submission Form The PSC Partners Registry keeps a map of healthcare providers submitted by Registry participants. By submitting a provider via this short form, Registry participants can help other people living with PSC to find a doctor who is familiar with PSC or who specializes in PSC care. For the Bereaved If you have a loved one who had PSC and has since passed away, whether due to PSC or not, you can create a "Person who has lost a loved one" profile to access this survey. Everyone with PSC is an important piece of the PSC puzzle, and their history is important to capture. We acknowledge and appreciate the generosity of those who have lost someone with PSC. The Registry has a wealth of other tools for you. To access these, you must create or log in to your Registry account. Detailed tutorials are available to get you started. View Summarized Survey Responses Registry participants can view a summary of other participants' de-identified responses to many survey questions. For instance, you can see the breakdown of age of PSC diagnosis across the whole Registry, or the number of Registry participants who have a family member living with PSC. This is available through the "Survey Analysis" tab in the navigation menu of the Registry dashboard. Health Info Tracking Basic Health Info: Log your current and past medications and supplements, treatments and medical procedures, labs/tests, and more. Symptoms and Activities: Log common PSC symptoms (or add your own) and activities such as exercise. Journal: Keep track of notes from doctor visits, prepare your questions to ask your doctor, or save notes on whatever else helps you keep up with your PSC care. Multiple Language Support The Registry Dashboard supports English, Spanish, French, Italian, and German languages. Surveys are currently only available in English; Spanish and French versions will be available later in 2025. Electronic Health Record (EHR) Import If your healthcare providers use an EHR system, the Registry platform supports importing data from many of the common EHR systems. You may select which records you wish to import into the Registry. To learn more about the Registry, please visit the Registry's Frequently Asked Questions (FAQ) . You can also contact the Registry team for any questions you have!

Publications Related to Clinical Trials in PSC Participants may receive emails from the Registry alerting them to clinical trial opportunities for which they may be eligible. Since the Registry will never release identifying information about a participant, it is up to each participant to contact the trial or study coordinators to learn more about the opportunity. PSC Partners encourages all patients to learn as much as possible when making decisions about joining a study or trial. Publications related to ongoing clinical trials can be found on this page. Please expand the section of interest by clicking on the title. PSC Partners Seeking a Cure recognizes the urgent need for treatments to stop or slow the progression of PSC. We support the development of clinical trials testing the safety and efficacy of new drugs for PSC, including the investigation of drugs currently given off-label. These publication resources are being provided to support patients and caregivers in making decisions about possible clinical trial participation. Please feel free to email us at registryresearch@pscpartners.org to suggest additional publications for consideration. Notes are added solely to assist in navigating the list. Readers are encouraged to read the full text to consider the full context of the evidence in the publication. Please see the PSC Partners Statement about the Use of Medications Not Approved by the FDA for PSC . PSC Partners is in the process of developing layperson educational materials on how to evaluate medical research, and a link will be added here soon. In the meantime, some aspects to consider: In what journal has this study been published? Is this a prominent journal with a peer-review process? Is this a less-prominent journal? Is this print or online? Is this an abstract from professional meeting proceedings (such as AASLD, EASL, NASPGHAN)? Typically this means a less rigorous review process than a journal article. Who is being studied? Adults? Pediatric? Both? With IBD or not? Stage of PSC? Pre-transplant or post-transplant? How large is the population being studied? The larger the population, typically the more generalizable the result. The more diverse the patient population being studied, the larger the study population needs to be in order to draw conclusions. Is there a comparator population? Is there a group against which the studied patients are compared? This could be a group receiving a placebo. Or this could be a group receiving an alternative treatment. Possibly the comparison is based on current understanding of the 'natural history' of disease progression. How long is the study? Is it prospective (defines patient cohort and collects data moving forward in time) or retrospective (uses data previously collected in the past)? Prospective cohort studies in general have higher accuracy (can decide exactly what should be collected and can pre-determine cohort) and retrospective cohort studies have higher efficiency (data already exists and cohort is limited by those on whom data exists). Chemomab: SPRING Study - CM-101 in PSC Patients Read More Escient Pharmaceuticals: PACIFIC Study - EP547 in Subjects With Cholestatic Pruritus Read More Mayo Clinic: Oral Vancomycin for PSC Read More Mirum Pharmaceuticals: VISTAS - A Study to Evaluate Efficacy and Safety of an Investigational Drug Named Volixibat in Patients With Itching Caused by PSC Read More Pliant Therapeutics: Phase 2a Evaluation of Safety, Tolerability, and Pharmacokinetics of PLN-74809 in Patients With PSC Read More