Odevixibat Shows Promise in Progressive Familial Intrahepatic Cholestasis
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Odevixibat Shows Promise in Progressive Familial Intrahepatic Cholestasis

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…

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TLMdX Accepts Late-Breaking Results on Odevixibat for PFIC
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TLMdX Accepts Late-Breaking Results on Odevixibat for PFIC

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.…

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New 5-Year Survival Data Available on Maralixibat for PFIC2

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…

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Phase 3 Odevixibat Clinical Trial Meets Endpoints for PFIC
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Phase 3 Odevixibat Clinical Trial Meets Endpoints for PFIC

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…

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Progressive Familial Intrahepatic Cholestasis Online Support Groups Provide Comfort During COVID-19

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…

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An ICU Nurse And Her Immunocompromised Daughter In The Age of Coronavirus

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.…

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“The Way That Everyone is Acting Now? Unfortunately, Thats Sort of How We Normally Run Our Lives.” A Immunosuppressed Patient And COVID-19.

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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How This Family is Raising Awareness for Progressive Familial Intrahepatic Cholestasis

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…

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“Stay at Home Right Now- Because My Kid Can’t” : A Mother Talks About COVID-19 and Her Immunosuppressed Daughter

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…

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After Facing Her Son’s Diagnosis, This Mom Started to Lead a PFIC Support Organization
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After Facing Her Son’s Diagnosis, This Mom Started to Lead a PFIC Support Organization

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…

Continue Reading After Facing Her Son’s Diagnosis, This Mom Started to Lead a PFIC Support Organization
Phase 3 Trial of Experimental Progressive Familial Intrahepatic Cholestasis Drug for Pediatric Patients Begins
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Phase 3 Trial of Experimental Progressive Familial Intrahepatic Cholestasis Drug for Pediatric Patients Begins

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…

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The First Ever PFIC Network Family Conference will Bring Families Affected by the Disease Together This Week

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…

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The Challenges Faced by Those Affected by PFIC Highlighted on Rare Disease Day
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The Challenges Faced by Those Affected by PFIC Highlighted on Rare Disease Day

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…

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New Development Deal Struck for Experimental Alagille Syndrome Treatment
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New Development Deal Struck for Experimental Alagille Syndrome Treatment

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…

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A Potential Drug for Progressive Familial Intrahepatic Cholestasis Achieves Fast Track Designation
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A Potential Drug for Progressive Familial Intrahepatic Cholestasis Achieves Fast Track Designation

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…

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An Experimental Treatment For PFIC Has Been Awarded Rare Pediatric Disease Designation by the FDA
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An Experimental Treatment For PFIC Has Been Awarded Rare Pediatric Disease Designation by the FDA

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…

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Phase 3 Trials for an Investigational Progressive Familial Intrahepatic Cholestasis Drug are Gearing Up
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Phase 3 Trials for an Investigational Progressive Familial Intrahepatic Cholestasis Drug are Gearing Up

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…

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Half of Children with PFIC will Receive a Liver Transplant by Age 10, Study Finds
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Half of Children with PFIC will Receive a Liver Transplant by Age 10, Study Finds

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…

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Opportunity is Coming for Your Child with PFIC!
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Opportunity is Coming for Your Child with PFIC!

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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