Friends of the PBC Foundation

11/06/2026

We had a powerful experience at the World Orphan Drugs Congress in Boston. Thank you to Rare Disease Advisor for inviting Danielle, the President of Friends of the PBC Foundation to speak on the panel "Beyond Awareness: Translating Lived Experience into Regulatory Data." The patient voice and patient experience should be at the forefront of all clinical trials.

Danielle said, "With primary biliary cholangitis, we have to be on some form of treatment to prevent our disease from progressing. With long-term placebos in confirmatory clinical trials, patients have to be off treatment and on placebos for 8 or more years risking liver cirrhosis, failure, and even death. If I was your mother, your sister, your daughter, your wife, or your friend, would you want me to be on a placebo that long?" The resounding answer in the audience was "No."

Hopefully regulators will begin to listen to the patients and utilize Real World Evidence as a way to bridge the gap when it comes to long-term placebos and patients living with rare disease.

11/06/2026

We are here in Boston for wonderful conversations hosted by Trialport regarding clinical trials and patients living with rare disease. Joana Pina, the President of CBP Portugal, our friends and partners, is speaking about her lived experience with primary biliary cholangitis. Thank you Keith Berelowitz for helping to put this together and making a huge difference for patients living with rare disease in the clinical trial space.

10/06/2026

Danielle, President of Friends of the PBC Foundation, will be speaking tomorrow at the World Orphan Drug Congress.

📍Boston, MA
Hall C : Track 1 - Rare Advocacy
🗓️ Tomorrow, June 11
⏰2:00-2:40 pm EST

Here is what she had to say, "See me tomorrow at World Orphan Drug Congress speaking about clinical trial regulations and the unethical challenges faced by patients living with primary biliary cholangitis. This is a very important topic to me since being the FDA PBC Patient Representative on the GIDAC committee regarding Ocaliva in September 2024. It is time for regulators to put the patient's lived experience first when it comes to regulatory data and standards. I will be speaking about the work that has come out of the International PBC Summit that the PBC Foundation, Friends of the PBC Foundation, and the PBC community as a whole across the globe have been working on for the last several years. It's clear that patients, patient advocacy groups, clinicians, biostatisticians, the pharmaceutical industry and all stakeholders in this community want an end to long-term placebo trials when it comes to PBC. Let's work together to make changes for the better in the rare disease community. Thank you Rare Disease Advisor for giving me this platform and an opportunity to be heard."

29/05/2026

The President of Friends of the PBC Foundation , Danielle, will be speaking at World Orphan Drug Congress in Boston, MA on June 11 on behalf of Rare Disease Advisor. The patient voice should be at the center of regulatory data and clinical trials. Hear from 3 patients with different rare diseases regarding regulatory data and the patients lived experience. Check out the event details and we hope to see you there!

https://app.terrapinn.com/event/world-orphan-drug-congress-usa-2026/planning/UGxhbm5pbmdfNDQ2MzAwNw==

28/05/2026

Stop by and see our partners at EASL for a chat about primary biliary cholangitis.

21/05/2026

Our President, Danielle, recently had the opportunity to share her experiences as a patient living with primary biliary cholangitis. Please check out her clips to see what it's like living with PBC. Thank you Health Union LLC for putting a spotlight on primary biliary cholangitis. It is estimated that over 100,000 Americans are living with PBC.

Watch "Real Talk," a video series on living with primary biliary cholangitis (PBC). Danielle and Lisa share how they manage unrelenting fatigue and symptoms.

16/05/2026

📍Portugal

Our partners CBP Portugal are hosting a meeting all about the fatigue patients experience living with primary biliary cholangitis. Fatigue can be so debilitating to patients.

Nem tudo o que parece cansaço é apenas cansaço.

A fadiga na CBP é um dos sintomas mais impactantes e menos compreendidos.
Neste encontro, o tema será abordado de forma clara e clínica pelo Prof. Doutor Filipe Nery.

Compreender é o primeiro passo para melhorar.

📍SRNOM - Norte Médico, R. Delfim Maia 405, 4200-256 Porto
🗓 16 de maio de 2026

👉 As inscrições ainda estão abertas
https://forms.gle/Lnzu8FJHMh4WiX6PA
A participação é gratuita, mas sujeita a inscrição prévia.

Evento promovido pela CBP Portugal, com o apoio científico da Associação Portuguesa para o Estudo do Fígado, Núcleo de Estudos das Doenças do Fígado da SPMI e Prof. Doutor Filipe Nery - Clínica do Fígado, e o patrocínio da Ipsen e da Gilead Sciences.

14/05/2026

We are excited for our partners and friends in the UK to have access to linerixibat. Severe pruritis can be such a devastating side effect for patients living with primary biliary cholangitis. All patients around the world deserve access to life-changing medications. 💜

Important news for the PBC community 💜

The MHRA has approved linerixibat for the treatment of cholestatic itch in adults living with PBC. This follows recent approval by the U.S. Food and Drug Administration (FDA), marking an important milestone for the global PBC community.

This approval represents an important step forward and offers real hope for people affected by this difficult symptom.

Our Head of Patient Services, Mo Christie, shared:
“Please do not underestimate the impact of PBC itch. It can be exhausting, isolating, and completely overwhelming at times. Having experienced severe itch myself, I know how desperately people have needed more effective treatment options. Seeing this news was incredibly emotional for me personally, because I know what this symptom takes from people and how long so many in our community have waited for progress in this area. This approval is an important moment and offers real hope for the future.”

You can read more, including what happens next regarding NHS access and NICE review, in our full article https://www.pbcfoundation.international/mhra-approves-linerixibat-for-the-treatment-of-itch-in-pbc/

03/05/2026

Don't forget that the next US Community Cafe is on Wednesday, May 6th! These virtual meetings are an incredible opportunity for patients living with primary biliary cholangitis to connect with other patients living with this rare disease. It's the perfect place to feel seen, be heard, and find support from others who truly understand the experience of living with PBC. Be sure to sign up and register with the PBC Foundation to have access to these inspirational virtual meetings!

Community Cafés are designed to help reduce isolation and bring people together in a relaxed, welcoming environment.

They provide an opportunity to connect with others impacted by PBC, share conversation and community and learn about the services and support available through the PBC Foundation.

In-person cafés are hosted by staff or volunteers, and online cafés are hosted by staff.
Here are the Community Café meetings coming up over the next week:

☕ Wednesday 06 May: US Online Community Café | 8am - 9am PT | 9am - 10am MT | 10am - 11am CT | 11am - 12pm ET
☕ Wednesday 06 May: Online Community Café | 6pm -7pm UK time

Visit our Events webpage for more information: https://www.pbcfoundation.org.uk/events/

You can read more about our café’s here: https://www.pbcfoundation.international/connecting-through-community-cafes/