NDA for Potential Progressive Familial Intrahepatic Cholestasis Treatment Cleared by FDA
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NDA for Potential Progressive Familial Intrahepatic Cholestasis Treatment Cleared by FDA

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.…

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Initiation of Global Phase 3 Trial of Alagille Syndrome Treatment
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Initiation of Global Phase 3 Trial of Alagille Syndrome Treatment

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,…

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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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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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NORD Announces Eleven New Grants for Rare Disease Research
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NORD Announces Eleven New Grants for Rare Disease Research

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…

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Experimental Treatment for Biliary Atresia Gets Orphan Drug Designation in the EU
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Experimental Treatment for Biliary Atresia Gets Orphan Drug Designation in the EU

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.…

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14-Year-Old Aidan Needs a Liver Transplant to Treat Rare Biliary Atresia Condition
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14-Year-Old Aidan Needs a Liver Transplant to Treat Rare Biliary Atresia Condition

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…

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