United Porphyrias Association

10/19/2026

Big news for our whole Porphamily, and especially our EPP and XLP community! This morning Disc Medicine announced positive Phase 3 results for bitopertin, an investigational treatment for erythropoietic protoporphyria.

To every patient and family who joined the Phase 2 and Phase 3 trials, and to every Phase 3 participant who completed the study and entered their data for one another: thank you. Research cannot happen without you, and it is because of you that bitopertin is headed back to the FDA for review.

This community cannot afford more delay. We urge the FDA to recognize the undeniable need in EPP and follow the data. Our community's voice echoes it: this treatment has changed lives and brought freedom. Together with our expert physicians, Disc, and regulators, let's get it to everyone who needs it.

Join us Wednesday at 6:00 PM ET for a community call with UPA and Disc leadership.

Register for link: https://www.porphyria.org/events/bitopertincall-phase3

Press release: https://ir.discmedicine.com/news-releases/news-release-details/disc-medicine-announces-success-all-primary-and-secondary

10/14/2026

One photo…so many stories…one incredible Porphamily💜🥹

WOW. What a weekend. What a community. What an unforgettable experience!

Looking at this photo, we see so much more than a group of people. We see strength, courage, friendship, understanding, and a whole lot of love🫂

Porphyria Palooza reminded us why we do what we do. Because if even ONE person walked away feeling less alone, more understood, or like they finally found their people… then every single moment was worth it.

To everyone who made this weekend possible, from our incredible patients and families to our volunteers, speakers, and UPA team: THANK YOU.

You are the heart of everything we do. And we couldn’t be prouder of this beautiful Porphamily🥹

Until we meet again!

💬 Now we want to hear from YOU! What’s one word that describes your Porphyria Palooza experience? Drop it in the comments! 👇

10/11/2026

and just like that… PORPHYRIA PALOOZA 2026 HAS COME TO AN END! 🥹

Our final day was filled with meaningful conversations, peaceful moments, and a whole lot of love.

We had sound bath, breakfast talk tables, Q&A with porphyria experts, closing reflections, and signing our Palooza yearbook… every moment reminded us how special this Porphamily truly is ✨

And then came the hardest part… saying goodbye ❤️‍🩹

To everyone who joined us, shared their stories, and made this weekend unforgettable: THANK YOU!

We may be leaving Kansas City, but the memories and friendships are coming home with us.

This isn’t goodbye. It’s see you later, Porphamily! 💜

P.S. Swipe all the way to the last photo for a very special goodbye from Simon, our cutest Palooza attendee! 🐾

10/11/2026

PORPHYRIA PALOOZA DAY 2 — PART 1! ✨

Today was all about the power of sharing our stories.

During our Voices of Porphyria session, patients with different types of porphyria opened their hearts and shared their journeys, challenges, life lessons, and words of wisdom.

There were tears, laughter, and so many moments that reminded us why this community is so special🫂

To every patient who bravely shared their story: THANK YOU. Your voice matters, your experiences inspire, and your courage reminds others that they are never alone💜

And trust us… Day 2 is FAR from over! 👀🎉

STAY TUNED FOR PART 2… WE HAVE A BIG SURPRISE COMING! 🤫

10/10/2026

DAY 1 OF PORPHYRIA PALOOZA WAS EVERYTHING!!! 🥹

Where do we even begin?! From the sweetest moments with Simon 🐶 to meeting patients with different types of porphyria, sharing hugs, making new friends, and reconnecting with our Porphamily! 🫂💜

Our UPA team has been working hard behind the scenes to make every moment special, and seeing everyone laughing, dancing, dressing up, and having the BEST time at the Game Show made it all worth it! 🏆😂

This is what Porphyria Palooza is all about: connection, community, and unforgettable memories.

And the best part? WE’RE JUST GETTING STARTED! 👀

We can’t wait to see what tomorrow brings! Who’s ready for DAY 2?! 🙌💜

10/09/2026

Warming up because these hugs aren’t going to give themselves! 😂💜

The UPA team is officially stretched, warmed up, and READY to welcome our Porphamily to Porphyria Palooza 2026! 🎉

We’ve been waiting for this moment, and we can’t wait for all the hugs, happy tears, laughter, and unforgettable memories ahead! 🥹

LET THE HUGGING MARATHON BEGIN! 🫂

Who’s ready for their first Palooza hug?! 👀

10/06/2026

Fishing tournaments, hunting, roping and riding horses were all part of growing up in Texas.
But behind those memories was something most people couldn’t see: Erythropoietic Protoporphyria (EPP).

Caul’s symptoms began around age 3. He remembers the pain initially feeling like “fire ants” and, as he got older, “a firecracker on the inside.”
During one fishing tournament, his hands turned purple and swelled to the size of a baseball glove after UV exposure. On family fishing trips, Caul and his older brother, who also has EPP, would sometimes dip their faces and hands into the ice chest trying to cope with the pain.

Still, Caul has refused to let EPP take away the things he loves.

His biggest advocate growing up was his mom, who worked to raise awareness about EPP before sadly passing away from brain cancer while Caul was in high school. Today, Caul and his brother are carrying her torch forward and keeping her cowboy legacy alive💜

Now Caul hopes to use his story to help others experiencing disability or discouragement experience the outdoors through hunting and fishing.

🔗 Read Caul’s full story through the link in our bio.

10/02/2026

October is here, and so are new chances to connect with our porphyria community! 💜

ConnectUps are small, peer-led groups on Zoom where patients and caregivers can share experiences, ask questions, and connect with people who truly understand. Whether you join every week or just once, there's a place for you.

👉 Check out this month's dates and find the ConnectUp that's right for you.

🔗 Register here: https://www.porphyria.org/connect-up

10/02/2026

📣 Bitopertin Expanded Access Program update!

Disc Medicine's Expanded Access Program (EAP) for bitopertin now has 8 sites open across the U.S., and more are expected:

📍 Boston, MA (2 sites)
📍 Cleveland, OH
📍 Detroit, MI
📍 Miami, FL
📍 New York, NY
📍Salem/Wilsonville, OR
📍 Winston-Salem, NC

Who may be eligible?
✔️ U.S. residents
✔️ Ages 12 and older
✔️ Diagnosed with EPP or XLP
✔️ Meet specific clinical criteria (NCT07603401)

The medication is provided at no cost through the program. Visits include 1–2 initial visits, a follow-up at about 3 months, and then visits every 6 months.

👉 Fill out the interest form to get updates and be contacted when a site opens near you: https://www.porphyria.org/features/bitopertineap

Completing the form does not guarantee enrollment.

Bitopertin is an investigational medicine and is not FDA-approved. Its safety and effectiveness have not been established.

Questions? Call 1-800-868-1292 or email [email protected] 💜

An Expanded Access Program, or EAP, for bitopertin is now available in the United States for people with EPP or XLP ages 12 and older. Complete an interest form to learn more.