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,…
According to a story from Healio, a recent study has illustrated that the drug denosumab can improve the bone density of patients with primary biliary cholangitis (PBC) and autoimmune hepatitis…
According to a story from Markets Insider, the biopharmaceutical company GENFIT recently announced that both the European Medicines Agency (EMA) and the US Food and Drug Administration (FDA) have awarded…
According to a story from BioPortfolio, the biopharmaceutical company Genkyotex has recently announced that the US Food and Drug Administration (FDA) has approved the company's Investigational New Drug (IND) application.…
According to a story from globenewswire.com, the biopharmaceutical company ContraVir Pharmaceuticals, Inc. has recently announced that its experimental drug CRV431 was able to successfully decrease scarring of the liver in…
Preclinical data supporting the efficacy of GKT831 as a potential therapy for cholestatic fibrosis has just been published in the Journal of Hepatology. GKT831 GKT831 is both a NOX1 enzyme and…
Researchers at GENFIT are excited about promising results from studies investigating a new potential therapy for PBC and NASH, two rare liver diseases. Primary Biliary Cholangitis Primary biliary cholangitis (PBC)…
The State of Rare Diseases in China The Illness Challenge Foundation (ICF) is an organization devoted to easing the burdens that rare disease patients living in China face. They accomplish this…
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