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…
The U.S. FDA has approved two treatments for people living with idiopathic pulmonary fibrosis (IPF). These therapies—Esbriet (pirfenidone) and Ofev (nintedanib)—both work to reduce fibrosis, or scarring, in the…
In a news release from late May 2022, pharmaceutical company SteroTherapeutics LLC, known for developing orphan drugs for rare conditions, shared that its novel therapy ST-003 received Orphan Drug designation…
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…
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…
Nobody wants to get sick on vacation; it can ruin a time meant to be fun and relaxing. Unfortunately, Kai McCulloch, a 13-year-old from Scotland, experienced this firsthand. They were…
The rare liver disease primary sclerosing cholangitis (PSC) may impact just .01% of people, but the extremely pernicious disorder's attacks on the liver mean bearers of the disorder account for…
Primary Sclerosing Cholangitis Primary sclerosing cholangitis (PSC) is a rare condition which causes inflammation within the bile ducts of the liver. It can lead to cirrhosis of the liver or…
Recently, clinical stage biopharmaceutical company Immunic, Inc. ("Immunic") has been working to develop potential treatment options for patients with primary sclerosing cholangitis (PSC), a rare condition for which no…
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…
Biological engineers at MIT are using a new technique to study various organs and their interaction with our immune system. According to a recent article in Science Daily, the researchers…
Primary sclerosing cholangitis (PSC) is a chronic liver disorder that typically affects males over the age of 30. That is why Di'Jon Thomas was so surprised to be diagnosed with…
Copyscape score: 2% Brigham and Women's is launching the nation's first ever center to conduct research on primary sclerosing cholangitis, a rare liver disease that affects about 30,000 people in…
According to a story from mirror.co.uk, a new invention called OrganOx has the potential to make liver transplants more effective and more widely available in the UK. A liver transplant…
According to a press release from the German-American biotechnology company Immunic Therapeutics, the Company recently began a proof-of-concept trial for its investigational primary sclerosing cholangitis treatment, IMU-838. About Primary Sclerosing…
According to a story from BioSpace, the biopharmaceutical company Immunic, Inc. has announced that the first patient has signed up for its proof-of-concept clinical study. This study is intended to…
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…
Primary Sclerosing Cholangitis (PSC) and Autoimmune Hepatitis (AIH) are both rare and chronic liver diseases. They cause the bile ducts in the liver to become inflamed, resulting in severe liver damage.…
According to a story from finanzen.at, the biotechnology company Orbsen Therapeutics recently announced that its immunotherapy product ORBCEL-C is to be tested in a multi-site clinical trial called MERLIN that…
According to a story from northjersey.com, Adam Levitz's life was hanging in the balance just a few weeks ago. His liver, which had endured years of damage from the rare…
Nonalcoholic fatty liver disease (NAFLD) is a condition which causes fat to accumulate in the liver. Nonalcoholic steatohepatitis (NASH) is a severe form of the disease in which the patient also…
Pliant Therapeutics, Inc., a biotechnology company specializing in developing and commercializing treatments for fibrotic diseases, announced on January 3, 2019 through PRNewswire, that it has initiated a Phase 1 first-in-human…
According to a story from The Straits Times, Nicole Lim, who recently turned eighteen, learned that she had three different rare diseases when she was just thirteen years old. Getting…
The United States Food and Drug Administration has awarded twelve research grants worth a total of over $18 million for research into rare diseases. For more information about this news,…
At 16 years old, I was told I would need a liver transplant within ten years. Being told at any age that you will eventually need a liver transplant is…
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