HHT Puerto Rico

BREAKTHROUGH: A treatment that targets the underlying cause of HHT—not just the symptoms—could be on the horizon.

This week, our close partners at Diagonal Therapeutics presented powerful new preclinical data showing that their antibody-based therapy can restore healthy blood vessel function and reverse key features of HHT in lab models.

Highlights from the findings:

• Restored ALK1-mediated signaling pathways
• Prevented and reversed arteriovenous malformations (AVMs)
• Improved survival and prevention of anemia in translational HHT models

🧬 Plus: The FDA just granted it Orphan Drug Designation, a special status that helps speed up the development of treatment for rare diseases

Why this matters:

DIAG723 – the first potential disease-modifying treatment for HHT -- is expected to enter clinical trials in 2026 and it is now backed by ODD incentives that can accelerate development.

We’re proud to help drive this progress forward—and even more proud to share it with our community.

Continue to support key patient initiatives and breakthroughs such as this by donating to Cure HHT today. Throughout the month of June your impact is doubled as the first $100,000 is match: curehht.org/donate

They told John he only had a month left to live...he knew he had to keep fighting - CureHHT John Bannon was told he had about a month left to live. For over a decade, John searched for relief to try to manage his increasingly severe nosebleeds. Bleeds so bad that he became known as “the nosebleed guy” to the local ambulance team – and so severe he was reliant on 2 to 4 units of blood...