APRIL UPDATE

Dear Friend,

Get ready to spread porphyria awareness, connect with community at upcoming events, participate in research and more! We are here to support you.

UPA is united with the global porpphyria community for an epic day of awareness. Come together with the international porphyria community on May 18, 2024 for Global Porphyria Day! Here's how you can get involved:

Turn Purple for Porphyria

On May 18th, join our porphyria friends from around the world to raise porphyria awareness by turning the world #PurpleforPorphyria!

• Get your purple awareness ribbon for a minimum donation of $5.
• Wear purple and encourage your friends to wear purple too!
• Light or decorate your home in purple
• Share your purple on social media with the hashtag #PurpleforPorphyria

Participate in the #MyPorphyria Awareness Campaign

Porphyria is so much more than a list of symptoms. Help others understand what it's really like.

Fill out and share your #MyPorphyria experience (don't forget to tag @UnitedPorphyrias)!

Get your fillable template and printable resources here.

Share #MyPorphyria videos and stories

In the week leading up to May 18th, watch for videos and patients stories, and share them with your networks. Let's raise porphyria awareness together!

Get your porphyria questions answered!

On Saturday, May 11 at noon ET, Dr. Amy Dickey of Massachusetts General Hospital will be joining us on Facebook Live to answer your porphyria questions.

Join the Porphyria Together Facebook group to join this session.

"Quantifying the impact of symptomatic acute hepatic porphyria on well-being via patient-reported outcomes"

Published in JIMD Reports

January 2023

Until now, most research about acute hepatic porphyrias (AHP, includes AIP, HCP and VP) has focused on symptoms and care during an acute attack.

In the latest Summing UP, we take a closer look at the POWER study which explored the full burden of AHP on patients from around the world.

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 Experts in Action page for more Summing UP

You're invited to a weekend organized by porphyria patients and families, for porphyria patients and families

Join us in downtown Chicago, IL from September 13-15, 2024 for weekend of connection, celebration and learning.

Program highlights include:

• Session to help you manage day-to-day on topics like advocating for yourself and nutrition
• Porphyria-specific information and Q&As
• An evening celebration featuring a music, dancing and games
• A patient-friendly environment with attention to light sensitivity, rest areas, safe foods, and immediate medical care access.

PorphyriaPalooza registration is totally free! Refundable deposit is required to reserve your spot.

Getting There: Travel Support Available!

UPA is pleased to offer a limited number of travel grants up to $500 to support travel to PorphyriaPalooza on a first-come, first served basis!

Eligible expenses include:

• Hotel accommodations
• Basic economy airfare
• Cost of a basic bus or train fare
• Ground transportation between your hotel and the airport/station
• Mileage to and from Chicago
• Car rental and gas cost
• Parking costs at hotel or departing airport/station
• 
  

PorphyriaPalooza registration is required to receive travel support.

The porphyria community is so fortunate to have passionate researchers, physicians and advocates who work alongside us to make a better future for porphyria!

In this feature we talk to our porphyria experts and advocates to learn about their work, promising research, new treatments and more!

Meet Craig Leppert from Shadow Jumpers

" I realized that my condition with the sun was either going to define my life or become a cool addition to the story of my life "

Can you introduce yourself and tell us a bit about your connection with porphyria?

My name is Craig Leppert, I have EPP and in 2017, I founded Shadow Jumpers which provides meaningful experiences and a sense of community to families suffering from sun-related conditions like porphyria.



I was born in Hawaii, grew up on the Jersey Shore and now I live in Los Angeles, so I like to say I've never done the easy thing when it comes to my life with porphyria. I grew up in a family that was very determined that we were going to have our own way despite our circumstances, and I think it’s made me more willing to be a little more aggressive in taking on life. It isn’t always easy, but I’m feeling very content with my life and disorder at this moment, which I think is the goal of a lot of people in our community.

Check out Shadow Jumpers!

UPA is proud to work with Shadow Jumpers to support kids and families with photosensitive porphyrias. Here are some of the great resources Shadow Jumpers provides:

• Camp Sun Escape
• UV clothing drive
• Shadow Jumpers comic book
• Family support to conquer your sun-related challenges
• Fun memes, merch and more!

New research from Dr. Amy Dickey, UPA Scientific Advisory Board member, was recently published in the International Journal of Dermatology.

The research found that wearing a dosimeter- a tool that measures light- could help track light exposure and predict if someone was at risk of developing EPP symptoms.

They study found that when the dosimeter was worn like a watch on the wrist, it could predict symptoms 78% of the time.

This technology could help porphyria patients keep track of light exposure over the day and help prevent reactions.

The United Porphyrias Association is looking for a part time Education and Community Engagement Coordinator!

This vital position is responsible for creating and managing educational programs and events aimed at both patients and healthcare professionals. This role is crucial for promoting knowledge, building a supportive community, and advocating for better care through engaging educational initiatives and events.

UPA's Kristen on Capitol Hill for the Coalition of Skin Diseases Hill Day

April 23rd was productive day on Capitol Hill!

UPA joined the Coalition of Skin Diseases to educate members of Congress about porphyria and advocate for:

• Increased NIH funding for rare disease research
• CDC funding for disease education and awareness
• Legislation for better access to treatments.

Learn more about CSD advocacy initiatives here.

Huge thank you to the Coalition of Skin Diseases for organizing!

International Congress of Porphyrins and Porphyrias : September 21-25, 2024

The ICPP in Pamplona, Spain, bringing together clinicians, researchers, scholars, patients and their families for three days networking and learning about the latest research in the porphyria field.

Learn more at the event website.

ICPP Patient Days

September 21, 2024 (in Spanish)

September 22, 2024 (in English)

Learn more about porphyria and emerging treatments, hear fellow patients' stories and learn about global advocacy initiatives.

Learn more at the event website.

Participant recruitment is now underway for a phase 3 study of dersimelagon, an investigational treatment for Erythropoietic Protoporphyria (EPP) and X-Linked Porphyria (XLP).

Fast Facts:

• First 3 sites opening soon in Boston, MA, Colombus, OH & Los Angeles, CA!
• Age 12+ with confirmed EPP/XLP diagnosis
• Have not previously received dersimelagon
• 150 participants needed globally
• The study drug is an oral medication
• Study includes an optional open-label extension

Interested or want to learn more? Fill out a confidential interest form.

Cimetidine in EPP and XLP Study

Patients with Erythropoietic Protoporphyria (EPP) or X-Linked Porphyria (XLP) are needed for an FDA-sponsored research study on cimetidine. The study will assess whether cimetidine (a medication normally used to treat gastrointestinal issues such as ulcers or acid reflux) can reduce protoporphyrin IX (PPIX) levels which may impact photosensitivity. 

Participating Study Sites: 

• Dr. Amy Dickey,  Massachusetts General Hospital 
• Dr. Karl Anderson, University of Texas Medical Branch 
• Dr. Herbert Bonkovsky, Atrium Wake Forest Baptist Health 

Fast Facts: 

• 3 study sites in the US 
• 20 total participants needed 
• Patients ages 15+ are eligible 
• Patients currently on Scenesse (afamelanotide) are eligible 

Of interest, a recent study in Denmark indicated the potential for cimetidine to lower protoporphyrin IX (PPIX) in patients with erythropoietic protoporphyria. See the research article here.

Longitudinal Study of the Porphyrias

In order to treat porphyria, you need to understand it. This large study is recruiting 1500 people with all types of porphyria to answer some important questions:

• What are the impacts of porphyria on quality of life and overall health?
• Why do some people with the same type of porphyria have more severe symptoms?
• How does porphyria progress over time?

The study findings will help develop new treatments and improve porphyria care.

Interested in participating? These are the eligibility requirements:

• Have been diagnosed with EPP, XLP, CEP, VP, AIP, HCP, PCT, HEP, or ADP
• Live in the US

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.

OR

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

Connect with your porphyria peers

Want to connect with others who understand what you're going through?

Connect UP groups are small, virtual peer-led groups for porphyria patients and caregivers.

Fill out an interest form to get matched with a group!

Join the Porphyria Together Facebook Group!

Did you know that UPA hosts a private Facebook group? Connect with and learn from your fellow patients, join special livestreams with doctors and more!

Join today!

 Add United Porphyrias INSTA, FACEBOOK, TWITTER to your social media feeds.