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.
PFIC Network nominated for Prix Galien Patient First Award
🎉 We've been nominated! PFIC Network has been nominated for the inaugural Prix Galien Patient First Award for our initiative IMPACT (Identifying research targets by Merging Patient And Clinician Treatment information).
This award, presented in honor of Michael J. Fox, recognizes initiatives embodying the principle "No decision for me without me" through patient-centered research, care, and advocacy that puts lived experience at the center.
Being named among this year's nominees is a huge honor and a reflection of the work our whole community of people living with PFIC, their families, clinicians and researchers have put into making sure the PFIC voice shapes PFIC research.
IMPACT was a two-year project funded by a Eugene Washington Engagement Award (EASO-30455) from the Patient-Centered Outcomes Research Institute.
Winners will be announced October 29 at the Galien Patient Summit in New York City. 🤞
Learn more about IMPACT and our ongoing work to build a PFIC research consortium by visiting https://impactroadmap.pfic.org/
Scoop of Comfort: Toddler with rare liver disorder inspires ice cream fundraiser for support foundation
"Ice cream might taste even sweeter when eaten for a good cause. Hailey and Skylar Medlin of Greenfield are hoping that's the case, as they host a Dairy Queen fundraiser next week to spread awareness about a rare liver disorder that has turned their 2-year-old daughter's life upside down. Rather than benefiting the family directly, all money raised at Wednesday's event will go to the PFIC Network."
Check out this news story about the Medlin Family by Shelley Swift in the Greenfield Daily Reporter
Results of IMPACT Published in Peer-Reviewed Journal
We are excited to announce the publication of "Identifying patient-centered outcomes in progressive familial intrahepatic cholestasis: Results from IMPACT" in the Journal of Pediatric Gastroenterology and Nutrition.
IMPACT (Identifying research targets by Merging Patient And Clinician Treatment information) was a two-year project (2023-2025) funded through the Eugene Washington PCORI Engagement Award (EASO-30455) program, an initiative of the Patient-Centered Outcomes Research Institute (PCORI). The project brought together PFIC patients, family members, clinicians, and researchers to build shared knowledge and tools for patient-centered comparative effectiveness research, a research approach designed to answer the questions that matter most to patients and families living with a condition.
The publication of these results marks an important milestone and a launching point. Building on the foundation IMPACT established, PFIC Network has received a new PCORI Engagement Award (January 2026–December 2027) to form a research consortium to plan future patient-centered comparative effectiveness research studies in PFIC.
Thank you to everyone involved—for your time, your trust, and your partnership in shaping the future of research in PFIC.
Severe itching in PFIC children disrupts sleep, family life: Registry analysis
Data from the PFIC Network Patient Registry is making news! Liver Disease News recently published an article highlighting the first peer-reviewed paper reporting findings from our registry, covering how pruritus impacts sleep, family quality of life, and the financial burden faced by PFIC families. It's a great plain-language overview of the paper for anyone who wants to learn more.
New PFIC Network PCORI Engagement Award
In our recent project, IMPACT, people living with PFIC, parents, doctors, and researchers learned together what patient-centered research means. We focused on how research can better address everyday challenges of life with PFIC. During this project, which was funded through the Eugene Washington PCORI Engagement Award program, an initiative of the Patient-Centered Outcomes Research Institute® (PCORI®), our community also shared the questions they would want research to address.
Now, we’re ready for the next step. We’re excited to share that PFIC Network has received funding for a new Engagement Award project.
Our new project, “Building Capacity to Engage in Patient-Centered CER on Progressive Familial Intrahepatic Cholestasis (PFIC),” will run from January 2026 to December 31, 2027.
Together, we will decide which research questions are most important to study first. These will become our PFIC research priorities.
We will also build a PFIC research group made up of people living with PFIC, parents, doctors, and research centers. This group - a patient-centered research consortium - will work together to plan future studies that follow these priorities. Community members like you will help guide the work every step of the way. We’re excited to share more soon and invite you to get involved as this project begins.
Help Fight Gigi’s Battle: Dover teacher looking for answers to solve toddler daughter’s rare liver condition.
"At first glance, one-year-old Giovanna “Gigi” Iachetta is a bubbly toddler with a radiant smile. But behind the giggles is a fierce little fighter living with Progressive Familial Intrahepatic Cholestasis (PFIC), a rare, incurable genetic liver disease affecting one in 100,000 children."
Check out Gigi's news story by Mariana Simioni on Dovernow.com!
2025 PFIC Charity Open & Labor Day Party
Local News Highlights PFIC Charity Disc Golf Event
A local news station recently featured coverage of the 3rd Annual PFIC Charity Open & Labor Day Party, held on September 1 in Campton, Kentucky. The event brought together disc golfers, cornhole players, and community members for a fun-filled day of competition, music, and celebration. In addition to raising critical funds for families affected by PFIC,the event also helped raise awareness — sharing our story with an even wider audience. We’re grateful for the continued support of our Red River Gorge community and proud to see our cause highlighted in the local news!
Their son has a rare disease: these parents want to raise awareness about the importance of organ donation
Their little one's case will one day require a liver transplant. Read the full story in Le Journal de Quebec!
“Itching and Scratching”, a Liver Disease News column about PFIC
Sophie Bourton is symptomatic carrier for progressive familial intrahepatic cholestasis and mother to a child with PFIC. Check out her column "Itching and Scratching" on Liver Disease News about the ups and downs of life in a family affected by PFIC.
New drug access for PFIC patients in Japan!
Exciting news for PFIC and Alagille syndrome patients in Japan! Maralixibat was recently approved by the Japanese Ministry of Health, Labour, and Welfare for the treatment of cholestatic pruritus. Congratulations to the Mirum Pharmaceuticals team!