Unfortunately, clinical studies do not always work out the way they’re expected to. Biopharmaceutical company Mirum Pharmaceuticals, Inc. (“Mirum”) learned this after the Phase 2 EMBARK trial failed to meet…
Clinical trials play a crucial role in better understanding rare diseases and therapeutic interventions. According to a news release from rare disease company Albireo Pharma, Inc. (“Albireo”), enrollment is now…
According to a recent article, a young boy who has been battling biliary atresia and was put on a liver transplant list has made miraculous progress. About Biliary Atresia Biliary…
Clinical trials are an important tool in medical research to understand more about the safety, efficacy, and tolerability of therapeutics - and how these may influence patient outcomes. Recently,…
When J.B. Lacombe's son was born, he showed signs of jaundice. Jaundice, caused by extra bilirubin in the blood, results in the yellowing of the skin, eyes, and mucous membranes.…
According to a recent article, a child with biliary atresia was able to celebrate her first birthday following her liver transplant. Biliary Atresia Biliary atresia is a condition occurring in…
If your life goes awry, do you trust your community to stand by you? Luckily, for 6th grader Ethan Chandler, his Carterville, Illinois community jumped in to help when he…
Marine Corps values include abiding by a code of integrity, respecting others, and respecting human dignity. For veteran John Rubino, this code of conduct helps shape the way he lives…
Over the last few years, more research has been dedicated to understanding the impact of gut health and the gut microbiome on overall health. More recently, researchers sought to understand…
For patients with biliary atresia, there are limited treatment options available. Currently, the rare liver and bile duct condition may progress to end-stage liver fibrosis and cannot be prevented or…
On June 24, 2020, Savanna Wrobel was born. She came a little early as a premature baby: 35 weeks and 3 days. But that didn't stop her parents, Wade and…
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.…
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,…
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…
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…
According to a recent press release from Albireo Pharma, the first participant has enrolled in the BOLD trial. The BOLD trial, which stands for Biliary atresia and the use…
According to a story from PR Newswire, the National Organization for Rare Disorders (NORD) has recently awarded a total of eleven new grant awards that will help fund essential research…
According to a story from Market Screener, the drug developer Albireo Pharma, Inc., recently announced that its investigational product candidate A4250 has earned Orphan Drug designation from the European Commission.…
Aidan Maddocks, 14, from Pietermaritzburg, was diagnosed with Biliary Atresia at birth, a childhood liver disease that only affects 1 in 10,000–15,000 live births. He was given a life expectancy…
Ryan Cole is a beautiful baby. You would never know from looking at him that he was struggling for life fighting a rare disease called biliary atresia, reported Courier Mail.…
It was a Christmas miracle for the Garcia family as their infant quickly recovered and was discharged from the hospital to enjoy the holiday at home, reported DailyMail. 1-year-old baby…
During a time when we get a lot of bad or polarizing news about the world, it's important to highlight the good stuff - so here is some good stuff…
Leave it to a sports award show to recognize what a real fighter looks like. ESPN's annual award show (the ESPYs) honored 15 year-old Jarrius Robertson last week with the…
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
