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BARE Inc. Launches First Biliary Atresia Patient Registry 

BARE Inc, a member of our council, has just released the first US-based patient registry for biliary atresia (BA). This registry focuses on consolidating crucial patient-generated data to advance research and treatments within the BA community. If you’re a BA patient or caregiver, visit now to register and contribute to this vital initiative.


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Potential New Treatment Option for Primary Biliary Cholangitis 

CymaBay Therapeutics has recently shared the findings from the phase 3 clinical trials of Seladelpar, designed for the treatment of primary biliary cholangitis (PBC). In these trials, Seladelpar demonstrated a notable reduction in biomarkers linked to adverse clinical outcomes, along with a concurrent improvement in pruritus, distinguishing it from existing therapies in the market. Pending approval, this therapeutic intervention holds the potential to significantly enhance the quality of life for individuals with PBC.

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Breakthrough Treatment Option for PSC Receives FDA Fast-Track Designation 

Chemomab Therapeutics has earned FDA fast track designation for its monoclonal antibody targeting primary sclerosing cholangitis (PSC). The antibody, known as CM-101 does double duty, fighting inflammation and fibrosis in key PSC pathways. The ongoing Phase 2 SPRING trial, assessing safety and biomarkers in PSC patients, is set to conclude in the second half of 2024. This designation expedites the development and review process for treatments addressing unmet medical needs for patients with PSC.

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GLI collaborates with Genfit to develop a plan for ACLF

At AASLD’s The Liver Meeting® in Boston, GLI collaborated with Genefit and accomplished hepatologists to comprehend the complexities and challenges inherent in developing treatment options for patients contending with acute-on-chronic liver failure (ACLF). Representing the patient voice, we plan to continue working closely with this team to chart the journey for ACLF patients and create an effective clinical trial process in the years to come.



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For more information about the Pediatric and Rare Liver Diseases Council or to learn more about joining, please visit our webpage or email