In a significant advancement for rare disease treatment in Japan, the Ministry of Health, Labour and Welfare (MHLW) has approved Bylvay® (odevixibat) for the management of pruritus associated with progressive…
Acknowledgment: This patient story is sponsored by Ipsen Biopharmaceuticals, Inc. and is promoted through the Patient Worthy Collaborative Content program. We only publish content that embodies our mission of providing…
Each week, in an email to his team, Albireo Pharma CEO Ron Cooper sends out the story of a child or family affected by conditions such as PFIC or Alagille…
In July 2022, Global Genes, a nonprofit 501(c)(3) organization and global advocate for those with rare diseases, awarded its Health Equity in RARE Patient Impact grants to members of…
Before you read on, make sure to check out Part 1 of our interview. In Part 1, Chris Peetz discusses his background, why he joined Mirum, and the process of evaluating LIVMARLI…
The American Association for the Study of Liver Disease (AASLD) held its annual Liver Meeting from November 4-7, 2022 in Washington, D.C. During the meeting, Mirum Pharmaceuticals shared two late-breaker…
October 5 is recognized each year as Progressive Familial Intrahepatic Cholestasis (PFIC) Awareness Day, a time to help spread awareness about this rare disease among the medical field and the…
According to a recent press release from Globe Newswire, Albireo Pharma announced that Lancet Gastroenterology published results of the PEDFIC 1 trial evaluating Bylvay® in the treatment of progressive…
According to a story from Globe Newswire, the rare disease company Albireo Pharma, Inc. has recently announced the release of new data from its phase 3 trials. These trials were…
Written by: Tyler Bradley, PFIC Network The PFIC Scientific Conference Held Many Great Presentations and Roundtables That Informed Both The Scientific And Patient Communities. Last month the PFIC Network hosted…
Recently, PFIC Voices released a guide for progressive familial intrahepatic cholestasis (PFIC) patients and families that will help them understand the condition and how they can get support. Progressive Familial…
It's no secret that drugs can sometimes be expensive, especially in regards to rare diseases or conditions. But how much would you guess a new drug is priced when…
In a video posted on Healio, Dr. Lorenzo D'Antiga from the Pappa Giovanni XXIII Hospital talked about the findings from a recent clinical study in which the drug odevixibat (also…
In a recent news release on Morningstar, biopharmaceutical company Albireo Pharma, Inc. ("Albireo") shared that its investigational drug candidate Bylvay (odevixibat) earned a positive opinion from the European Medicines…
According to a press release from the rare liver disease company Albireo Pharma, Inc., the US Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for odevixibat.…
According to GuruFocus, the European Medicines Agency (EMA) recently validated a Marketing Authorization Application (MAA) submitted by biopharmaceutical company Mirum Pharmaceuticals, Inc. ("Mirum"). The MAA was centered around maralixibat, an…
During the Association for the Study of Liver Diseases (AASLD) Liver Meeting, which took place from November 13 to 16, biopharmaceutical company Albireo Pharma ("Albireo) announced data from the Phase…
According to a story from GlobeNewswire, the rare disease liver company Albireo Pharma, Inc., recently announced positive results from a phase 3 study testing the company's experimental drug odevixibat as…
Later this week, the American Association for the Study of Liver Diseases (AASLD) will hold its virtual The Liver Meeting Digital Experience (TLMdX). Altogether, TLMdX will last from November 13-16.…
This year, the Digital International Liver Congress took place online from August 27-29, thanks to COVID-19. During the conference, biopharmaceutical company Mirum Pharmaceuticals announced data from its Phase 2 INDIGO…
Recently, biopharmaceutical company Albireo Pharma announced positive results from a Phase 3 clinical trial. The trial, PEDFIC 1, studied the safety, efficacy, and tolerability of odevixibat for patients with progressive…
Claire Brinkley Claire Brinkley's daughter Eleanor is diagnosed with progressive familial intrahepatic cholestasis (PFIC) which is a rare disease affecting the liver. As a newborn, she appeared perfectly normal. But at…
When Trey Kearns was two, he had a terrible itch. His itch wasn’t like yours - it was inside the body, meaning it's an itch you can’t scratch. They came…
Emily Ventura was already well aware of the risk of germs before coronavirus. She works as a nurse in the ICU and takes care of her 8-year-old, who is immunosuppressed.…
Brooke Ramirez's pregnancy with her first daughter went as planned, and her new baby Trinity passed her developmental milestones. It wasn't until she was five months old that they noticed…
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