The PFIC Newsroom

Every story we have to tell brings us one step closer to finding more treatment options for PFIC. Whether we are raising disease awareness, advocating for our families, inspiring hope or coming together at an event, PFIC families are working tirelessly to make themselves known. The PFIC Newsroom page highlights stories that have been published outside of the PFIC Network. If you have been in the news or media and would like your story to be featured on this page, please contact us.

Note: PFIC Newsroom stories are not endorsements or prescriptions of any treatment either available or in development. If you are interested in learning more about if treatment options are the right choice for you or your child, please discuss with your doctor.

Itch
Exciting news for PFIC patients in Europe!
July 8, 2024

Exciting new for PFIC patients in Europe!  The European Commission has granted marketing authorization for maralixibat for the treatment of progressive familial intrahepatic cholestasis in patients three months of age and older. 

View Mirum’s press release to find out more!

Itch
FDA Approval of LIVMARLI (maralixibat) for the treatment of cholestatic pruritus in PFIC
March 14, 2024

Congratulations to the Mirum Pharmaceuticals team! Yesterday they received FDA approval for LIVMARLI as a treatment for cholestatic pruritus associated with PFIC. We are so excited for PFIC patients in the US to have another tool in their toolbox to fight back against the terrible itch of pruritus. A big thank you to the Mirum team for their hard work & vision and to all the clinical trial participants who made this day possible!

View Mirum’s press release!

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Project REACH Across the Globe
November 10, 2022

This July, PFIC Network was honored to receive a Global Genes Health Equity in RARE Patient Impact grant to address challenges affecting underserved patient communities. 

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PFIC Scientific Conference: Wrap-Up and Next Steps
June 1, 2022

The PFIC Scientific Conference Held Many Great Presentations and Roundtables That Informed Both The Scientific And Patient Communities.

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Research Announcement: The PFIC Network Research Grant Program is Now Open
December 20, 2021

Thanks to Our Amazing Community, the PFIC Network Has Been Able to Follow Through with Scientific and Research Priorities to Start the PFIC Network Research Grant Program by the End of the Year.

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Cincinnati Children’s Launches Research Center for Rare Liver Diseases
December 13, 2021

Cincinnati Children’s Hospital is investing in research of cholestatic liver diseases such as PFIC.

CZI Awards $13 Million to Patient-Led Organizations Advancing Rare Disease Research
November 3, 2021

PFIC Network has partnered to work as a patient led collaborative research network, being experts on our own disease, to facilitate research programs for PFIC.

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Bespoke Gene Therapy Consortium to Fund Research into Improving Standardizing Methods for Preclinical Testing of Gene Therapies.
November 1, 2021

The NIH, FDA, and FIH have unveiled a public-private partnership aimed at accelerating gene therapies for the roughly 30 million Americans living with a rare genetic disease.

News
FDA Approval of Bylvay (Odevixibat) for the treatment of Progressive Familial Intrahepatic Cholestasis
July 20, 2021

We are excited to hear that the FDA has approved Bylvay (Odevixibat) for the treatment of Progressive Familial Intrahepatic Cholestasis.

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European Commission Approval of Bylvay (Odevixibat) for the treatment of Progressive Familial Intrahepatic Cholestasis
July 19, 2021

We are excited to hear that the EMA has approved Bylvay (Odevixibat) for the treatment of Progressive Familial Intrahepatic Cholestasis.