April UPDATE

Dear Friend,

We understand that living with porphyria goes far beyond medical care! This month, we’re highlighting some of the many ways people in our community nurture well-being, build connection, and help move research forward. In this eNews, you’ll find opportunities to engage, be inspired, and stay connected.

• Raising Awareness through Art: Join the Art Challenge
• Save the Date: Porphyria Palooza is coming up this fall!
• Get ready for Global Porphyria Day: Order your awareness ribbons today!
• LEVEL UP with Sun Escape: Can you spot the differences?
• Get Involved in Research: New opportunities to support porphyria research
• Advancing Research: American Porphyrias Expert Collaborative
• Treatment Updates: Updates on dersimelagon and bitopertin for EPP & XLP
• Porphyria Voices: Physiotherapy and the Road to Recovery
• Our Stories: Meet Scarlett and Brady
• Connect UP with Your Community: New Connect UPs and monthly meeting schedule

Read on, friends. We’re glad you’re here. 💜

With gratitude,

The UPA Team

In honor of Global Porphyria Day, we invite the global porphyria community to help tell the story of porphyria through art. Whether your medium is visual, written, digital, performance-based, or something entirely your own, we would love to see your creativity in action.

Here are the challenge details:

• Open to anyone in the porphyria community, including patients, caregivers, advocates, doctors and researchers.
• All forms of creative expression are welcome, including visual, digital, literary, performance, design, and experimental work.
• One submission per participant.
• AI use is prohibited, except for translating original work into English
• Entries must be submitted by Sunday, May 17, 2026. 
• Voting begins on Global Porphyria Day, May 18 and ends June 1.
• Top three entries will receive a prize:
• 1st: $1,000
• 2nd: $750
• 3rd: $250

Full rules, submission form and details available at porphyria.org/art-challenge!

Thank You to Our Art Challenge Sponsor!

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

Get ready for a Porphamily Reunion!

Porphyria Palooza is coming up this fall! Join us for a weekend that is all about connection, learning and fun with your porphyria community.

Date: October 9-11, 2026

Location: Kansas City, MO

Travel support will be available. Registration, hotel and event details coming soon!

On May 18, 2026 we'll be joining our porphyria friends from around the world to raise porphyria awareness for Global Porphyria Day!

Here are some ways you can get involved:

Turn the World Purple for Porphyria



• Order your free porphyria awareness ribbon today!
• Invite your family, classmates and colleagues to wear purple, and light or decorate their homes and workspaces in purple

Participate in the Art Challenge



• Submit an art piece for the International Porphyria Art Challenge by May 17.
• Vote for and share your favorite pieces beginning May 18- Global Porphyria Day!

Share your #OneThing



• Help others understand porphyria by answering the prompt "One thing I wish you understood about porphyria..."
• Look for resources and tips to help you get started soon!

Thank You to Our Global Porphyria Day Sponsors!

The countdown is on, and Sun Escape energy is building!

UPA and Shadow Jumpers will welcome 32 photosensitive campers, their families, and 45 incredible volunteers to Victory Junction for an ALL FUN, NO SUN long weekend. Altogether, we’ll be hosting nearly 200 people for a special weekend of sun-safe fun, connection, and community.

This year’s theme is LEVEL UP! - this celebrates the imagination, games, and the creative ways our campers make the time before sunset full of fun and possibility. In the spirit of the weekend, here’s a game for you! Can you spot all 8 differences between these two scenes? 👀

Did you find all 8 differences? Email info@porphyria.org with your answers and we’ll send you a little treat!

New 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. 

Stay tuned for more information about Portal Therapeutics’ upcoming clinical trial in EPP and XLP.

Seeking participants who do not have porphyria

UPA invites friends and family members of people with porphyria to help advance porphyria research.

Some studies need volunteers who do not have porphyria, called controls, so researchers can compare results. These volunteers are matched to participants based on factors such as age, sex, race and ethnicity, general health, and sometimes skin tone.

UPA is currently identifying potential matches for future studies. Complete the interest form to be considered. If you may be a match, UPA will contact you before sharing your information with a research site.

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.

Advancing porphyria research and care takes close partnership between the expert and patient communities

APEX, the American Porphyrias Expert Collaborative, brings together the leading porphyria physicians and researchers in the field, while UPA helps ensure that patient voice, lived experience, and community priorities remain central to that work. Together, APEX and UPA help strengthen research, education, and care across the porphyrias.

What makes someone a porphyria expert?

APEX expert membership requires a high level of demonstrated specialization and contribution in the porphyrias, including meaningful involvement in research, publications, scientific presentations, leadership in clinical care, and sustained work advancing the field. APEX members also lead Porphyria Centers of Expertise, serve on the UPA Scientific Advisory Board, and help educate and consult with treating physicians.

APEX also recognizes lived experience as an essential form of expertise and works closely with UPA to ensure patient and family perspectives help shape progress.

Where are Porphyria Centers of Expertise?

Porphyria Centers of Expertise across the United States provide specialized care, informed treatment guidance, educational outreach, and, in some cases, access to research studies and clinical trials.

APEX Awards $500,000 in International Postdoctoral Grants

APEX recently announced the recipients of the 2026 APEX International Postdoctoral Fellow Grants. Five $100,000 grants were awarded to support mentored postdoctoral research in heme biosynthesis and the porphyrias. The selected projects reflect the innovation and momentum needed to move porphyria research forward.

Congratulations to the recipients and their mentors!

• Cindy Ament, PhD (University of California, San Francisco, Mentor- Bruce Wang, MD)
• Lenore Yalom, PhD (Massachusetts General Hospital, Mentor- Amy Yeung, MD)
• Rory Tinker, MD (Icahn School of Medicine at Mount Sinai, Mentor - Manisha Balwani, MD)
• Shahriar Sohel, MD (Icahn School of Medicine at Mount Sinai, Mentor - Makiko Yasuda, MD, PhD)
• Sonali M. Vaidya, M.Sc. (University of Bonn, Germany, Mentor - Diana Imhof, PhD)

Positive Results From Phase 3 INSPIRE Study of Dersimelagon for EPP & XLP Presented at AAD

In March, Amy (Dickey) Yeung, MD (MGH) presented encouraging Phase 3 results for dersimelagon, an investigational oral treatment for people living with EPP and XLP, at the American Academy of Dermatology.

Speaking on behalf of Tanabe Pharma America and the study investigators, Amy (Dickey) Yeung, MD (MGH) shared key findings and information of the INSPIRE study:

• Participants taking dersimelagon were able to spend more time in sunlight before feeling early warning symptoms, and other study findings also pointed in a positive direction.
• The study included 165 adults and adolescents ages 12 and older with EPP or XLP.
• Researchers also reported that the treatment was generally well tolerated, with most side effects described as mild to moderate.

We are deeply grateful to the patients and families who took part in this research! Your time, trust, and willingness to participate help move the field forward for everyone living with porphyria.

Disc Medicine Planning an Expanded Access Program for Bitopertin

Disc Medicine is planning an Expanded Access Program, or EAP, for bitopertin in the United States for people with EPP or XLP ages 12 and older. An EAP is a way some patients may be able to receive an investigational treatment outside of a clinical trial and before regulatory review. Patients will need to meet specific criteria to be considered.

This program is not open yet. To help us understand interest and share updates as more details (such as study sites, inclusion criteria, etc.) become available, we invite you to fill out this interest form.

Porphyria Voices is a UPA blog where we share experiences, insights, and advice from the porphyria community. Discover valuable knowledge, foster connections, and join us in raising awareness to empower and unite our porphyria family.

Finding Strength in Small Steps: 
A Patient's Guide to Recovery from Paralysis

"It's better to take a day to breathe and rest than to face future days full of frustration, extreme tiredness, and anxiety."

By Maria Paula Galvis,

My first days in the ICU were painful and filled with so much uncertainty. The pain was often so bad it didn't let me think or even be fully conscious of my situation, but I do remember worrying that I would never be able to walk again. Every day in the hospital, I wondered when it would end, when I’d be able to walk and move through the world with ease. Deep down, I believed that recovering from paralysis wouldn't be too complicated, maybe a couple of months, like when the effect of something just wears off.

Have you experienced paralysis?

Connect with others who understand what you're going through. UPA's next Acute Porphyrias & Paralysis Connect UP is Thursday, May 14.

Meet Scarlett, AIP Warrior

"My hope is simple: that no other patient will have to nearly die to be believed."

My name is Scarlett. I was 23 years old when my porphyria journey began. I had just gone through a divorce and was raising two young children on my own- one was four, the other just one. I was trying to survive, rebuild, and keep going, but I was overwhelmed. Emotionally, physically, financially.

Then, the perfect storm hit. I was following a very strict no-carb diet. And I witnessed a traumatic car accident where a young girl died. That trauma, combined with extreme stress and dietary restriction, unknowingly created the conditions to bring my porphyria out of dormancy.

About two weeks later, I became very sick.

Starting in May, Scarlett will host a monthly Sound & Restore Connect UP to help you slow down, release tension and reconnect with yourself. Learn more and sign up here.

Meet Brady, EPP Warrior

"What might sound small to others feels extraordinary to me. Walking from my car into a grocery store without thinking about it. Sitting in a classroom without worrying about sunlight reflecting off a whiteboard or someone’s laptop onto my skin."

My name is Brady Wheeden. I am 20 years old, and I have lived with Erythropoietic Protoporphyria (EPP) my entire life. This condition doesn’t just affect your life, it completely changes it and everything that you do. 

Before bitopertin, my life was defined by the sun. I could only spend about 5 to 10 minutes outside before I would feel a reaction begin. And when it hit, it wasn’t just discomfort, it felt like my skin was being held up to a burning stove while being stabbed with pins and needles at the same time. It was intense, overwhelming, and something most people could never truly understand. 

EPP didn’t just limit me, it controlled me. Everyday things that most people don’t even think twice about, like walking from your car into a store, going to class, or hanging out with friends outside, were major challenges. I had to constantly plan my life around avoiding sunlight. Even making friends in college became difficult because I couldn’t casually walk to class with people or go to outdoor events. I always had to think about where the sun was, how strong it was, and how quickly I needed to get out of it. 

Brady shared his story to help raise awareness and advocate for access to bitopertin. Share your story and get involved at patientsforbitopertin.org!

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.

Raising Awareness at Digestive Diseases Week

UPA will be attending Digestive Disease Week (DDW) 2026 in Chicago from May 2-5. DDW is a major global conference for professionals working in gastroenterology, hepatology and related fields.

New Awareness Messaging and Materials

UPA will be debuting new awareness messaging and materials for the meeting with the message Is It Porphyria? Our goal is to encourage healthcare professionals to consider porphyria as a potential diagnosis when patients present with unexplained, recurring, or hard-to-connect symptoms including abdominal pain, nerve pain, fatigue, dark urine, liver problems, and phototoxicity. Greater awareness can lead to earlier diagnosis, more informed care, and better outcomes for patients.

Porphyria Presentations

Two porphyria-related programs will be featured at DDW:

• Acute Hepatic Porphyria: A Case-Based Patient Journey
  Presented by Brendan McGuire, MD and Bruce Wang, MD and Kristen Wheeden, DrPH including a patient case presentation featuring Mayra Martinez
• Chronic Symptoms Reported by Patients with Acute Hepatic Porphyria in the POWER Study in a Subgroup Analysis of Nonrecurrent Patients. 
  Presented by Kristen Wheeden.

We're pleased to share two new Connect UPs that focus on our mental, physical and spiritual well-being.

Sound & Restore

First Thursday of each month at 1:30 PM ET

Join us for a gentle ConnectUp session led by Scarlett Dias, designed to help you slow down, release tension, and reconnect with yourself. Through sound healing, Scarlett will guide you into a calming space where your mind and body can rest, reset, and restore.

Reflection & Prayer

Third Tuesday of each month at 7:00 PM ET

Join us for a special ConnectUp centered on prayer, reflection, and spiritual support, led by Shawn Willis. This gathering offers a peaceful space to come together, share intentions, and find comfort in community. Whether you’d like to pray, listen, or simply be present, all are welcome.

Upcoming Connect UP Meetings

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

Cutaneous Porphyrias Connections- This Sunday!

Monthly on the 4th Sunday of the month
Hosted by Candace

Next meeting: Sunday, April 26 at 3 PM ET

Location: Zoom- register for the link

Acute Porphyrias (AIP, HCP, VP & ADP)

Weekly on Wednesdays

Hosted by Mayra and Ginger

Next meeting: Wednesday, April 29 at 1:30 PM ET

Location: Zoom- register for the link

CEP & HEP Connections

Monthly on the 1st Saturday of the month

Hosted by Justin

Next Date: Saturday, May 2 at 6 PM ET

Location: Zoom- register for the link

Sound & Restore

Monthly on the 1st Thursday of the month

Hosted by Scarlett

Next Date: Thursday, May 7 at 1:30 PM ET

Location: Zoom- register for the link

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

Hosted by Geri

Next meeting: Thursday, May 14 at 1:30 PM ET

Location: Zoom- register for the link

Porphyria Caregivers Connect UP

Monthly on the 3rd Saturday of the month

Hosted by Wilson and Charles

Date: Saturday, May 16 at 12 PM ET

Location: Zoom- register for the link

AHP Men (AIP, HCP & VP) Connect UP

Monthly on the 3rd Sunday of the month

Hosted by Geoff

Date: Sunday, May 17 at 6 PM ET

Location: Zoom- register for the link

Reflection & Prayer Connect UP

Monthly on the 3rd Tuesday of the month

Hosted by Shawn

Date: Tuesday, May 19 at 7 PM ET

Location: Zoom- register for the link

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.