June UPDATE

Dear Friend,

As we move into a new season, we're feeling especially grateful for this incredible porphyria community. Thank you for helping create a world where no one faces porphyria alone.

We’re excited to share this month’s updates, opportunities, and stories with you. Read on for:

• Upcoming Events! Webinars on new AHP guidelines and EPP/XLP investigational treatment results, Patient Forum in Dallas, and Porphyria Palooza!
• Art Challenge Winners Announced: Check out the top entries!
• New Online Training for Health Professionals: Share with your care team.
• Research Opportunities: Your participation makes a difference.
• UPA in the Community: Speaking at the FDA
• Partner Updates: APEX advocates for better testing, latest activities from UPA Latam.
• Our Stories: Meet Camila and Hailey.
• Connect UP with Your Community: find the groups for you!

We're glad you're here💜

With gratitude,

The UPA Team

PSST...Ever wonder what the story behind the UPA logo is? Read to the end to find out!

Webinar: Let's Talk About the New Acute Porphyria Guidelines, June 25

Join porphyria experts Manisha Balwani, MD, MS of Mount Sinai and Hetanshi Naik, PhD of Stanford University to learn more about the new guidelines for AIP, HCP & VP.

We'll be diving into the recommendations and exploring how they apply to you.

• Date: Thursday, June 25
• Time: 6:00 pm ET
• Location: Zoom- register for link

Q&A to follow the presentation.

In-Person: Patient & Family Forum in Dallas, TX on June 27

Join UPA's Kristen Wheeden and Mayra Martinez, and a panel of knowledgeable health care professionals, including Scientific Advisory Board members Karl Anderson and Taha Bat, for a day all about porphyria.

Connect with others in your community and learn about your porphyria.

This patient- and family-focused day of learning will cover all types of porphyria and will include discussion about porphyria causes and triggers, managing symptoms, liver health, patient stories and resources to support you on your porphyria journey.

• Date: Saturday, June 27, 2026
• Time: 10 am to 2 pm
• Location: UT Southwestern, Dallas, TX

Lunch provided. No cost to attend, but please register in advance.

In-Person: Porphyria Palooza, October 9-11!

Porphyria Palooza is coming to Kansas City, MO this fall! Join us for a weekend that is all about connection, learning and fun with your porphyria family.

Join us for the most amazing weekend with your Porphamily!

Porphyria Palooza is all about the joy and power of coming together. The Palooza Weekend agenda is packed with education session on topics you want to learn about, opportunities to connect and tons of fun activities!

Registration is $25/person for adult and free for children and teens under 18, and includes all meals and snacks, a dance party, special outing and more!

Porphyria Palooza is an accessible, family-friendly event. Get all the details and register at porphyriapalooza.com.

Webinar: Understanding New Research in EPP on June 27

Join Portal Therapeutics for a patient-friendly breakdown of the Phase 2a GATEWAY study data for investigational PORT-77, and learn what’s next for EPP and XLP research.

📅 June 27, 2026 | 11 AM ET (find your local time)

How to Attend

Registration is not required and patient information is not being collected. To attend, add the event to your calendar and click on the link on June 27 (link).

Want a reminder? Sign up for an email reminder here.

PORT-77 is an investigational agent and has not been approved by any global health authorities.

UPA is pleased to share the results of the International Porphyria Art Challenge!

With more than 3,700 votes cast and and 30 entries in crochet, poetry, painting, drawing, stained glass, film, collage, photography, digital illustration and more- the challenge helped highlight and share the realities of porphyria with the world.

First Place: I thank you lots, Porphyria by Maria Paula Galvis Forero

Second Place: 4 ever Porphyria Strong by Brayden Hamilton

Third Place: Overcoming pain to bloom again by Margarita Maria

Thank you to everyone who participated. All submissions are now featured on the art challenge page and we will continue to share and highlight these works throughout the year!

Thank You to Our Art Challenge Sponsor!

The International Porphyria Art Challenge was made possible by the support of Recordati Rare Diseases.

We are pleased to share some valuable new online education resources are available for health care providers. As a patient or caregiver you may also find them helpful, though some of the language is technical.

Acute Hepatic Porphyria: A Case-Based Patient Journey

Through this program, learners will be better able to diagnose AHP in a timely manner so that patients can receive evidence-based treatment as part of patient-centered care. Featuring porphyria experts Dr. Bruce Wang and Dr. Brendan McGuire, and porphyria patient Mayra Martinez.

Up to 1.00 AMA PRA Category 1 Credit(s)™

Maximum of 1.00 ABIM MOC points

EPP and XLP in Practice: Addressing the Evolving Therapeutic Landscape and Real-world Patient Needs

In this online, on-demand educational session, two expert clinicians, Dr. Rebecca Karp and Dr. Raegan Hunt discuss common and under-recognized symptoms, systemic complications, and updated guideline-recommended care. for the protoporphyrias: EPP & XLP.

Up to 0.75 AMA PRA Category 1 Credit™

UPA Brings the EPP Patient Voice to the FDA

Kristen gives remarks at FDA.

On June 4, UPA President Kristen Wheeden spoke at the FDA Commissioner’s National Priority Voucher meeting on bitopertin, an investigational treatment for EPP and XLP being developed by Disc Medicine.

Her remarks emphasized that patients must be meaningfully included in regulatory decision-making for rare, serious, and complex diseases.

Kristen highlighted the significant burden of these diseases, including severe light-induced pain, loss of daily activities, delayed diagnosis, and the need for additional treatment options. She noted the EPP community’s long-standing engagement with the FDA and urged regulators to consider disease burden, scientific evidence, biomarkers, and patient experience when evaluating treatments.

UPA is supporting Patients for Bitopertin, a patient-led advocacy initiative to support access to bitopertin for patients with EPP & XLP.. Learn more and get involved: patientsforbitopertin.org

APEX Advocates for Improved Porphyria Testing in USA

This month Molecular Genetics and Metabolism published recommendations related to porphyria testing in the United States by members of American Porphyrias Expert Collaborative (APEX).

APEX recommends that specific tests should be available to all physicians in the US for diagnosis and monitoring of porphyrias, and that harmonization of laboratory practices and results reporting across laboratories should be achieved. Check out the publication.

UPA is working with APEX to improve information about and access to testing. Look for new resources coming soon!

UPA Latam Update: Building Connections and New Group Meetings!

UPA Latin America continues to build momentum across the region, both through direct patient support and through stronger connections with local communities! Some recent highlights include:

• A visit to Argentina in May for the Patient Day organized by Dr. Valeria Pachioli. It was wonderful to connect with the community there and hear directly from patients and families about the challenges they face, particularly around diagnosis, access to specialists, and knowing where to turn for reliable information.
• Starting monthly community meetings to create more spaces for Spanish-speaking patients to connect, share experiences, ask questions, and feel less alone. This is something we want to continue building for both patients and caregivers since many of them in the region still do not have regular access to others who understand what living with porphyria can be like.

How do you use AI? UPA wants to know!

Healthcare providers, institutions, and organizations want to support patients and families in using AI in safe, helpful, and meaningful ways. This short, anonymous survey will help us better understand those needs.

What we learn could help guide future education, increase awareness, and support the development of practical tools such as prompting guides, disease-specific chatbots, and other helpful resources.

Observational Study for EPP & XLP

UPA is supporting the patient community and clinical study sites to assist in patient referrals for stEPP, a clinical outcomes assessment study (also referred to as an observational study) that tracks the disease and its impact on individuals living with Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP). This study is sponsored by Portal Therapeutics.

Participation involves study-related visits with clinicians experienced in your condition and contributes to future research. Your participation in stEPP will help inform the design of future clinical studies, including those involving investigational therapies. 

Porphyria patients are the center of all porphyria 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.

Meet Hailey, VP Warrior

“I often think about all the people who have had to fight to be believed. The people whose pain was minimized. The people who were told they were too young, too different, too rare, too complicated.”

I'm 20 years old. I live with Variegate Porphyria (VP), a rare genetic disease that many people have never heard of. I'm queer. Most days, I carry all of these realities at once.

My porphyria journey began when I was on a trip to Puerto Rico visiting family. While I was there, I got extremely sick. That was when the vomiting started. When we got back home to Florida, I still couldn't keep food or drinks down. At that point, I also couldn't use the bathroom.

Meet Camila, EPP Warrior

“One of the most difficult things about EPP is missing out.”

My name is Camila Baerwald, I’m 37 years old, originally from Chile, and I now live in Los Angeles. I got married four years ago to my husband, Yarden, and together we have two wonderful children: Yosef Mendel, who is 2 years old, and Anael Shira, who is 11 months old.

I love sports, especially squash and Pilates. I also enjoy singing, dancing, being creative through art, trying new things, and spending time with friends and family.

My journey with Erythropoietic Protoporphyria (EPP) began when I was just 3 years old. I remember one of my earliest symptoms perfectly. I was at the beach and told my mom, “Mami, I’m burning.” 

We want to help you 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.

Connect UPs are safe, friendly online meet ups where you can connect with others who understand what you're going through.

Find the Connect UP Groups for You!

Connect UP groups are formed based on interest or shared experiences. Follow the links below or visit the Connect UP page to learn more about upcoming groups!

For Acute Porphyrias (AIP, HCP, VP & ADP)

• Weekly Acute Porphyrias
• Youth
• Men
• Paralysis

For Cutaneous Porphyrias

• CEP & HEP
• All Cutaneous (EPP/XLP, PCT, CEP, VP, HCP)

For Well-Being, Not Porphyria Specific

• Reflection & Prayer
• Caregivers
• Pacientes de porfiria
• LGBTQIA+

New Connect UP Groups

Pacientes de porfiria

Únete a nuestra próxima reunión Conéctate, un espacio seguro y acogedor para personas que viven con Porfiria (cualquier tipo de porfiria)

Moderada por Mayra Martínez, paciente con PAI, esta reunión brinda la oportunidad de conectar con otras personas que realmente entienden el camino de vivir con porfiria. Regístrate.

LGBTQIA+ Connect

Connect and celebrate with fellow LGBTQIA+ members of porphyria community. Hosted by Elsie, Tommy and Ollie.

First meeting: Monday, June 22 at 6 PM ET

Register

Connect Online Anytime: Porphyria Together Facebook Group

Share your successes and struggles on our private Facebook community: Porphyria Together.

This group is a great place to connect with others, gain tips and insights on managing your porphyria, and access exclusive livestreams where porphyria experts take your questions.

What's the story behind UPA's logo? Professor Porphy explains!

(click to enlarge)

Do you have a question for Professor Porphy? Send it to info@porphyria.org!