August UPDATE

Dear Friend,

UPA is growing - in initiatives, staff, and space! We’re pleased to introduce an affiliate organization, our newest team member and our new office location.

In this eNews, you'll learn more about these exciting developments, as well as:

• Join the conversation: Staying active with acute porphyria
• We're gathering as a community: Join us for the Patient & Family Weekend, October 11-12
• Summing UP: new research on the burden of EPP in daily life
• Meet our newest team member: Keili McEwen, Legislative Advocacy Manage
• Patient story: Courtney on growing up with EPP
• Porphamily Reunion recap: an afternoon of fun with our porphyria family
• Research opportunities: get involved in porphyria research

... and so much more!

August 2025 memories

We are committed to bringing you the connection, information and resources you need, and this new growth expands our horizons and brings us closer to our goal of a world free from the pain and challenges of porphyria. Always remember that you are not alone 💜

Warmly,

The UPA Team

Staying fit when you have acute porphyria

Whether your goals are training for a race, recovering from an attack or are just trying to move more, staying fit when you have porphyria is a balancing act. 

Join Ironman athlete and AIP patient Lauren Thompson for a discussion on staying active when you have porphyria. We'll be sharing strategies on nutrition, listening to your body and more.

Date: Thursday, August 28, 2025

Time: 7:00 PM ET (find your local time)

Focus will be on acute hepatic porphyrias (AIP, HCP, VP).

Sign up to get the zoom link.

We are counting down the days until we can see you in person!

The Patient & Family Conference in Arlington, VA is a weekend for you! As an event organized by patients, for patients and families, everything is focused on creating a space where you can connect, learn, and feel truly understood in a safe and supportive environment.

Sessions include:

• Patient Stories
• Q&A with porphyria experts
• Tips and strategies for managing day-to-day
• Harmonizing your health care
• Updates on from industry partners on treatments
• Mental health and taking care of yourself

We'll also have lots of time for you to connect with your porphyria friends, including ice breakers, games and a Saturday night dance party!

Registration is $25 per family. All sessions, meals and activities are included!

When?

Saturday, October 11 at noon- Sunday, October 12, 2025 at noon

Where?

Westin Arlington, Arlington, VA

Need help getting there?

$250 travel stipends are available. Learn more and apply here.

Explore the latest science and research

The 2025 International Porphyrias Symposium, October 9-12, is a premier educational event dedicated to advancing knowledge in heme biosynthesis and the pathogenesis and treatment of the acute hepatic and erythropoietic porphyrias.

Patients and caregivers are able to attend the Symposium at a discounted rate. Learn more!

We are thrilled to announce the launch of a new affiliate organization- Porfirias Unidas Latinoamerica (United Porphyrias Latin America)!

Porfirias Unidas Latinoamerica is a network built by and for patients that truly understands what it means to live with porphyria in Latin America, and will complement existing initiatives in this region.

Check out all the new resources in Spanish at porfiria.lat and follow @porfiriasunidas on Instagram!

Together, we can make a world free from the pain and challenges of porphyria. 💜

United Porphyrias anuncia el lanzamiento de una nueva organización afiliada — Porfirias Unidas Latinoamérica (United Porphyrias Latin America)!

Porfirias Unidas Latinoamérica es una red construida por y para pacientes, que comprende profundamente lo que significa vivir con porfiria en Latinoamérica, y complementará las iniciativas existentes en esta región.

Descubre todos los nuevos recursos en porfiria.lat y sigue a @porfiriasunidas para acceder a contenido sobre porfiria en español. 

Estamos muy emocionados de trabajar con UPA Lat. ¡Juntos, podemos construir un mundo libre del dolor y los desafíos que trae la porfiria! 

Meet Estephany Escarcena, UPA Latin America Founder

"There’s something powerful about finally putting a name to what you’re going through. "

When I was a child, I dreamed of doing something that would make a real difference in people’s lives. Back then, I didn’t know how or when it would happen, or what shape it would take. I just knew I wanted my life to matter beyond myself. 

Years later, after a series of confusing symptoms and several visits to hospitals, I came accross a TikTok video from Katri Parra, that video ended up changing everything. It led me to my porphyria diagnosis, a moment that, while scary, also felt like a kind of relief. There’s something powerful about finally putting a name to what you’re going through. It gives you a direction, a sense of control. Sometimes, having an answer (even a tough one) is better than endless uncertainty.

But more significantly, it introduced me to a global community of patients: people who, like me, were navigating a complex and often misunderstood condition. Among them, I found many from Latin America, where the need for resources, support, and connection felt even more urgent.

"The Burden of Erythropoietic Protoporphyria (EPP): Results From the EPP Life Impact and Genetic Health Trajectory (LIGHT) Study"

This study asked adolescents and adults with erythropoietic protoporphyria (EPP) or X-linked protoporphyria (XLP) how sunlight affects their skin, daily life, emotions, school and work, and healthcare use.

The goal was to put numbers to what many already live every day. This type of research is essential to help advocate for treatments and improved access to care by helping policy makers, physicians and researchers understand the needs of patients.

Summing UP features the latest porphyria research in easy-to-understand summaries that have been reviewed and approved by the UPA's Scientific Advisory Board of porphyria experts.

Visit our Spotlight Porphyria blog for more Summing UP.

We are excited to introduce Keili McEwen as our new Legislative Advocacy Manager.

Keili brings more than 20 years of experience at the intersection of public policy, politics, and strategic advocacy in Oklahoma, Arkansas, and Washington, DC. Over the course of her career, she has advised governors and legislators, led winning campaigns, built coalitions, and achieved policy victories at every level of government.

She has held leadership roles in government, including serving as Director of Public Affairs for Governor Mary Fallin and in the office of Governor Mike Huckabee, served as Vice President of Government Affairs at a large, non-profit Oklahoma financial institutions. Most recently, she founded The Auctor Group, a government relations and public affairs firm based in Oklahoma City.

Beyond her professional expertise, Keili brings a deeply personal connection to porphyria advocacy. In 2015, her husband, Colin, nearly lost his life to Acute Intermittent Porphyria (AIP). Since then, she has been a devoted caregiver and passionate advocate for patients and families navigating porphyria. Keili shares:

“I am thrilled to work with UPA in a legislative advocacy capacity. This is a really special moment for me. My husband, Colin McEwen, has bravely battled AIP for many decades. For years, I stood by his side as a caregiver and I’ve seen up close the challenges and the resilience this community carries. I hope I can make a lasting difference, for him, and for all affected by porphyria.”

Keili lives in Oklahoma City with Colin, their son, and a lively household of pets.

We are honored to welcome Keili to the UPA team and look forward to her leadership in strengthening the patient voice on Capitol Hill and in state legislatures across the country.

Make sure lawmakers hear the porphyria community loud and clear

Keili will be working closely with patients and families to make sure lawmakers hear the porphyria community loud and clear. If you’re interested in sharing your story or taking part in upcoming advocacy opportunities, please reach out to us at info@porphyria.org.

The United Porphyrias Association's team is growing and we need more space. Our new office will accommodate our team as we expand the programs and resources we offer to the porphyria community!

Our new office is next door to our partner organization, the American Porphyrias Expert Collaborative, allowing us to work closely together to create a world free from the pain and challenges of porphyria.

You'll now find us at : Suite 400W, 7315 Wisconsin Avenue in Bethesda, MD.

Meet Courtney, Living Fearlessly with EPP

“I wouldn’t change how I dealt with it, we figured it out as we went and did the best with what we knew. BUT I would change how others treated me, judged me, and picked on me.”

I started showing symptoms at the age of 2 during an end of summer beach day with my family. My hands and feet swelled up so bad the skin cracked so where you could almost see bone. The doctor said I was probably stung by a jellyfish and we went on with life until spring rolled around and I had another reaction. I was lucky to not have a lengthy process for diagnosis, however, the only answer we received was, “I read this in a textbook while I was in college, but don’t know much about it. Here’s your daughter, just keep her out of the sun.” at this point is was 1991, so we couldn’t google anything and were left to figure it all out on our own.  

My tolerance is about 15 minutes of sun before the warning signs of my skin tingling and stinging sets in. Knowing I only have a few minutes to completely escape the sun before a full blown reaction starts, at that point causing me to burn from the inside out, like I have a chemical burn underneath my skin.

Want to share your story?

We're here to help! Fill out an interest form or reach out to our Community Engagement Team at info@porphyria.org.

We had a great time at the Porphyria Palooza Reunion on August 16 with our Porphamily!

It felt incredible to connect with friends- new and old- over several competitive rounds of games like What is the Price?, Wheel of Prizes and Friendly Feud with John from Grin Events.

While the competition was fierce, Team Blue, ended the afternoon victorious and win bragging rights until we face off again!

Porphyria patients are at the center of all porphyria research! Your participation is essential to gaining a better understanding of porphyria, developing new treatments and improving diagnosis and care.

Phase 3 Clinical Trial of Bitopertin for EPP and XLP

Are you or your child 12 years or older and diagnosed with erythropoietic protoporphyria (EPP) or X-linked protoporphyria (XLP)? If so, an exciting study may be an option for you!

Disc Medicine is conducting the APOLLO Study to evaluate Bitopertin, an investigational oral medication designed to reduce protoporphyrin IX levels and potentially ease symptoms related to sun exposure.

Key Details:

• Participants needed at U.S. study sites- new sites are open in Seattle, WA and Galveston, TX
• Travel & related expenses covered by the study sponsor

If you're interested in learning more, please fill out the Study Interest Form—your responses are completely confidential and only used to determine interest in receiving further details.

Have questions? We’d love to talk! Call 800-868-1292 or email info@porphyria.org for more information.

Short-Term Clinical Trial Opportunity for Adults with EPP

The GATEWAY Phase 2 Study is enrolling adults with Erythropoietic Protoporphyria (EPP) to test a potential new treatment.

Some Details:

• The study takes place in Phoenix, Arizona
• Travel, hotel, and meals are covered for you (and a caregiver/travel partner, if wanted)
• US and international participants welcome
• 9 Days at the study site + travel days
• Compensation provided for your time

You may qualify if you:

• Are 18 or older with a confirmed EPP diagnosis (EPP only)
• Experience symptoms within 40 minutes of sun exposure (can be on an approved drug, cannot be in another clinical trial)
• Are a non-smoker
• Other inclusion/exclusion criteria will apply

To learn more: Reach out to us at info@porphyria.org.

Survey Opportunity for Acute Hepatic Porphyrias (AIP, HCP & VP)

An interview study is underway to understand patient experiences with acute hepatic porphyria.

We are looking for individuals ages 15 and over in the United States or Canada who have acute hepatic porphyria (AHP).

As part of this study, you will complete a one-on-one approximately 90-minute telephone or videoconference interview scheduled at a time that works well for you, and you will later fill out three online questionnaires that take approximately

20 minutes to complete.

You will receive $150.00 USD or Canadian equivalent as compensation for your

time to participate.

Please note, you/your child may not be eligible for the study if you/your child: have taken

GIVLAARI (givosiran) or hemin prophylaxis within the past six months; reside in Alberta,

Saskatchewan or Newfoundland and Labrador; are between the ages of 15-17 and reside in

Quebec; or have certain medical conditions.

Get involved in research!

There are multiple studies going on right now that need your patient expertise. Check out our current opportunities and find out how you can participate here.

OR

contact UPA at 1-800-868-1292 or info@porphyria.org for more information

Did you know there is an online community of 2,000+ people impacted by porphyria for you to connect with?

UPA's Porphyria Together Facebook Group is a active and growing space for patients and families to connect, ask questions and support. each other.

The group also hosts live Q&A's with porphyria experts and provides opportunities for you to shape the resources that UPA offers to you!

 Add United Porphyrias to your social media feeds. 

You made it to the end! 🎉

Thanks for making your way through August UPdates! We're so glad to have you with us.

Take a moment to click through the links and buttons above to find helpful resources, ways to get involved, and opportunities to stay connected.

As always, we’re here for you - supporting, listening, and moving forward together.

With gratitude,

The UPA Team