Shannon and Todd Palmatier have three children, two of which have progressive familial intrahepatic cholestasis (PFIC). Their sons' diagnoses have brought the lack of knowledge and advocacy to the attention…
Emily Knakmuhs's two-and-a-half-year-old Kennedie had just made it past a tough couple of years. When she was born, her family noticed she wasn't growing, but her pediatrician attributed it to…
The Calgary Herald published a story about 2 and 1/2 year old Armando Perez's rare disease Progressive Familial Intrahepatic Cholestasis (PFIC), with has left him unusually itchy. It's so important…
According to a story from delmarvanow.com, a nine year old boy named Trey Kearns has progressive familial intrahepatic cholestasis (PFIC) a rare disease that affects the liver. The symptom that…
According to a story from drugs.com, the biopharmaceutical company Mirum Pharmaceuticals recently announced that they have begun dosing the first patient in a phase 3 clinical trial. This clinical trial…
Emily Ventura has never met in-person another person living with progressive familial intrahepatic cholestasis (PFIC), her daughter’s life-threatening ultra-rare genetic disease. That will change on June 21st when Emily and…
According to a story from Financial Buzz, the biopharmaceutical company Albireo Pharma, Inc. issued a statement on Rare Disease Day which emphasized the company's continued commitment to progressive familial intrahepatic…
According to a story from prnewswire.com, the drug developer Mirum Pharmaceuticals recently announced that is has entered an agreement with Shire which grants exclusive rights for marketing and development of…
The United States Food and Drug Administration has granted Fast Track designation to a drug being researched as a potential treatment for progressive familial intrahepatic cholestasis, a serious liver disease…
The U.S. Food and Drug Administration has granted the experimental drug A4250 rare paediatric disease designation for the treatment of progressive familial intrahepatic cholestasis. The full article can be found…
According to a story from Financial Buzz, the pharmaceutical company Albireo Pharma recently announced that the first patient has been enrolled in its Phase 3 trial testing its experimental candidate…
An international team of researchers studying the effects of progressive familial intrahepatic cholestasis (PFIC) in children have found that half of the children had received a liver transplant by age…
Albireo Pharma is in the midst of preparing for an exciting Phase 3 clinical trial to test A4250 for the treatment of progressive familial intrahepatic cholestasis (PFIC). PFIC is a…
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