My name is Brenda. In April 2021, I received a life-changing diagnosis of Primary Biliary Cholangitis (PBC), which I attribute to divine intervention. The journey began in January 2021, when…
In 2003 at the age of 38, I was diagnosed with primary biliary cholangitis, or PBC. I went for routine bloodwork which came back showing crazy high liver function and…
It has been almost a decade since the European medical community welcomed news of an innovation for primary biliary cholangitis (PBC). Therefore, the EC’s recent conditional approval of Ipsen’s drug…
Mirum Pharmaceuticals has long been a leader in the rare liver disease space, driving forward with a mission of empowering patients and families, and identifying novel therapeutic interventions to help…
To the primary biliary cholangitis (PBC) community - it's time to rejoice! For the first time in nearly one decade, the U.S. Food and Drug administration (FDA) has approved a…
What’s better than being in warm and sunny Arizona? Getting to learn alongside the amazing primary biliary cholangitis (PBC) community while there! The PBCers organization held its 2024 Patient Education…
We can all say that our mothers gave us the gift of life. But how many can say that they gave the gift of life in return? One teenager in…
In mid-May 2023, Pharmaceutical Technology reported that a new combination treatment had earned Orphan Drug designation. This combo? Obeticholic acid (OCA) and bezafibrate. Together, they represent a potential therapeutic…
Cognitive deficits have been recognized as symptoms of advanced liver disease for many years. As reported in Bioengineer, the American Journal of Pathology recently published a study providing insights…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
Gannex has just announced that they have dosed the first patient in their Phase 2 clinical trial examining ASC42 as a treatment for those diagnosed with primary biliary cholangitis (PBC).…
Albireo Pharma Inc. has just announced the results from their Phase 1 clinical trial for A3907, an ASBT inhibitor. The study produced positive results, achieving its primary and secondary endpoints. Researchers…
Acknowledgment: This story is sponsored by CymaBay Therapeutics and is promoted through the Patient Worthy Collaborative Content program. We only publish content that embodies our mission of providing relevant, vetted and valuable information…
In a news release from mid-December 2021, rare liver disease company Albireo Pharma, Inc. ("Albireo") shared that positive topline data was available from a Phase 1 clinical trial evaluating A3907…
In a story posted on msn.com, primary biliary cholangitis patient Cathy Mumford talks about why she feels it is important for patients living with a rare disease to participate in…
In the European Union (EU), Orphan Drug designation is granted by the European Medicines Agency (EMA). The status is given to drugs or biologics intended to treat, prevent, or diagnose…
Previously, Ocaliva (obeticholic acid) was approved as a prescription treatment for patients with primary biliary cholangitis (PBC), a rare and chronic liver disease. However, Market Screener reports that biopharmaceutical company…
According to a story from Medscape, US Food and Drug Administration (FDA) has recently issued a new restriction on the use of obeticholic acid (marketed as Ocaliva). The agency now…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
Albireo has recently initiated their Phase 3 ASSERT trial, according to GlobeNewswire. This study will evaluate odevixibat as a treatment for Alagille syndrome. This therapy fits well with Albireo's mission,…
A recent study sought to evaluate the impact of family history on the risk of autoimmune diseases, particularly primary biliary cholangitis and autoimmune hepatitis. There hasn't been much research conducted…
CymaBay Therapeutics, a biopharmaceutical company based in Newark, CA, develops and provides access to novel therapies with a recent focus on primary biliary cholangitis (PBC). The company is currently developing…
CymaBay Therapeutics has recently released the results from ENHANCE, a study of seladelpar as a treatment for primary biliary cholangitis (PBC). These results were positive, proving that this medication has…
According to a story from GlobeNewswire, the biopharmaceutical company CymaBay Therapeutics, Inc. recently announced encouraging topline findings from a phase 3 clinical trial. This study was testing seladelpar as a…
Enanta Pharmaceuticals has recently released the results from the second phase of its INTREPID trial, which studied EDP-305 as a treatment for primary biliary cholangitis (PBC). The results were positive,…
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