UPdates


PHASE 3 INSPIRE STUDY FOR MT-7117 NOW RECRUITING PARTICIPANTS
Eligible participants must have EPP or XLP diagnosis, have not previously received MT-7117, be age 12+ and meet additional eligibility requirements. Please complete an INTEREST FORM. Your response is completely confidential, only for designating your interest in further information. Contact UPA at 800-868-1292 or email us at info@porphyria.org with any questions.
 

PHASE 2 AURORA TRIAL FOR BITOPERTIN TO TREAT EPP IS NOW FULLY ENROLLED!
Thank you to Disc Medicine, all the patients, supportive family and friends, researchers and study coordinators who have made this trial possible.

 

MT-7117 PHASE 3 TRIAL CONTINUES ACCESS TO DERSIMELAGON AT 200MG DOSE

 

Patients who completed the Phase 3 clinical trial and are currently participating in the Open Label Extension (OLE) will now have continued access to dersimelagon.

  • Who is eligible? Patients who completed the Phase 3 clinical trial
  • Extension time period: Participants will have access to up to an additional 30 months
  • Action: Contact your Study Site to transition to this dose at your next regular study visit.


PRELIMINARY FINDINGS RELEASED FOR BITOPERTIN
Disc Medicine recently shared preliminary findings from its ongoing, Phase 2 open-label BEACON trial in Australia which is evaluating bitopertin in patients with EPP and XLP. Early results found:

  • Bitopertin consistently decreases protoporphyrin IX
  • Patients had significant increases in reported sunlight tolerance and quality of life
  • Bitopertin was well-tolerated with no serious adverse events, no reported discontinuations or dose reductions, no adverse events greater than Grade 1 (mild), and no meaningful changes in observed in mean hemoglobin level

These data were presented at the European Hematology Association 2023 Congress in Frankfurt, Germany. More details about the results are available HERE.

UP and Coming

Q&A WITH PORPHYRIA EXPERT- RECORDING NOW AVAILABLE

View the latest live Q&A with a porphyria expert and get support from your peers on the Porphyria Together Facebook Page! WATCH THE RECORDING!
 

SUN ESCAPE WEEKEND FOR FAMILIES

Shadow Jumpers and the United Porphyria Association are teaming up for SUN ESCAPE WEEKEND 2024 happening at Victory Junction in North Carolina from May 3-5, 2024. 

This transformative weekend will bring together 20 kids, ages 6-16, and their families from around the country to celebrate all things Fun and all things No Sun. LEARN MORE AND APPLY!

 

 

PORPHYRIAPALOOZA 2024: INAUGURAL PORPHYRIA PATIENT WEEKEND

Porphyria is hard enough… let’s get together to share stories, build community, and have fun!

Mark your calendar for Porphyriapalooza 2024: Inaugural UPA Patient Weekend on September 13-15, 2024 in Chicago, IL

Stay tuned for details!

 

YOU ARE NOT ALONE!Connecting with others who understand what you're going through is a great way to build a support system and community. UPA is pleased to launch Connect Up small group meetings for patients and caregivers to connect and socialize with others in a safe and inclusive environment. Connect UP groups will be peer-led and offered regularly over Zoom. SIGN UP HERE.
 

PORPHYRIA TOGETHER ON FACEBOOKWe welcome you to join Porphyria Together, a new closed Facebook group where you will find helpful information in a safe community setting. JOIN US

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Types of Porphyria

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Acute Intermittent Porphyria (AIP)

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Hereditary Coproporphyria (HCP)

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Variegate Porphyria (VP)

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ALAD-Deficiency Porphyria (ADP)

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Porphyria Cutanea Tarda (PCT)

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Erythropoietic Protoporphyria (EPP) and X-Linked Porphyria (XLP)

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Our Mission

The United Porphyrias Association is committed to improving the quality of life of the porphyria patient community and is relentlessly focused on advancing disease awareness, research, and therapies in all the porphyrias.

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A world where patients are free from the pain and challenges of porphyria.

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Member Highlight

Victor Mejias

Erythropoietic Protoporphyria (EPP)

"I was diagnosed at the age of 6 with EPP after my parents noticed that every time I was outside I was experiencing anguishing discomfort. I never fully comprehended my diagnosis until I was able to connect with others. With the help of the UPA, I want to be able to help others live more comfortably with EPP and support the vision of a world where patients are free from the pain and challenges of porphyria. "

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