Achievements & Milestones
Hereditary hemorrhagic telangiectasia (HHT) went from a condition with no organized field to one with accredited centers, international guidelines, and therapies in trials. This is the record of how.
Then and now
What has changed
Within living memory, a person with HHT could go an entire lifetime without a correct diagnosis, and a clinician who suspected it had nowhere to send them. There were no accreditation standards, no agreed screening protocols, no registry, and no treatments aimed at the underlying mechanism.
All four of those things now exist. None of them appeared on their own. Each was built by patients, families, clinicians, and researchers who decided that a condition affecting more than two million people should not remain invisible.
Where the progress has been
🏥
A network of expert care
More than 55 HHT Centers of Excellence now operate worldwide, each accredited against defined standards for multidisciplinary care. A family with a diagnosis can find a team that has seen the condition before.
📘
International clinical guidelines
Consensus guidance for diagnosis and management now exists and is periodically updated, which means care no longer depends on which clinician a patient happens to see.
🧬
The genetic basis identified
Pathogenic variants in ENG, ACVRL1, and SMAD4 account for the large majority of cases. Families can now confirm a diagnosis and test relatives definitively.
💊
Therapies aimed at the mechanism
Treatment has moved beyond managing each lesion after it bleeds. Agents that act on abnormal blood vessel formation are now in clinical use and in trials.
🗃️
Research infrastructure
A patient registry, a biobank, and agreed outcome measures now exist, which is what allows a trial in a rare disease to be designed and powered at all.
🏛️
Recognition in policy
HHT is named in rare disease policy and research funding conversations where it was previously absent, largely because patients showed up and asked for it.
🤝
A community that finds each other
Families who once believed their nosebleeds were a private oddity now have a name for the condition and a community that understands it without explanation.
1991 to today
The full record
Every milestone since the foundation was established, searchable and filterable. Use the chips to see only one kind of progress, or search for a center, a trial, or a year.
| Year | Milestone | Kind |
|---|---|---|
| 2026 | Global Phase 3 HEROIC study initiated | Therapeutics |
| 2026 | First patient dosed in the DIAMOND trial | Therapeutics |
| 2026 | HHT-specific eligibility secured in the FY2026 Peer Reviewed Medical Research Program | Advocacy |
| 2024 | PATH trial results published in the New England Journal of Medicine | Therapeutics |
| 2024 | NIH-sponsored PATH trial becomes the first positive, large-scale study in HHT | Therapeutics |
| 2024 | FDA grants Fast Track designation to the Vaderis allosteric AKT-inhibitor VAD044 for HHT | Therapeutics |
| 2024 | First positive industry-led trial for HHT announced by Vaderis Therapeutics, with Cure HHT support | Therapeutics |
| 2024 | Diagonal Therapeutics secured $128 million in Series A funding for its HHT program, helped by Cure HHT | Therapeutics |
| 2024 | Centralized biorepository launched, giving researchers nationwide access to HHT tissue | Research |
| 2024 | HHT Connect, the first global patient registry, rolled out to the community | Research |
| 2024 | Seed grant provided to the Broad Institute of MIT and Harvard to study precision therapy | Research |
| 2024 | Federal funding protected for a third year, contributing to a 44% increase in new patients seen at Centers of Excellence | Advocacy |
| 2024 | Congressional Brief held inside the US Capitol as part of HHT Capitol Hill Day | Advocacy |
| 2024 | 15th International HHT Scientific Conference hosted, with more than 300 attendees | Community |
| 2024 | National Patient and Physician Conference hosted outside Philadelphia, setting attendance records | Community |
| 2024 | First in-person HHT Continuing Education event held in Nashville | Community |
| 2024 | HHT Kids Day hosted in partnership with UCSF in San Francisco | Community |
| 2024 | UC San Diego Health re-certified as an HHT Center of Excellence | Care network |
| 2022 | Federal funding secured for US HHT Centers of Excellence for the first time, after more than 15 years of advocacy | Advocacy |
| 2022 | First Cure HHT Research Network convening held in Boston, producing the Research Roadmap | Research |
| 2022 | Therapeutic Development Arm of Cure HHT created | Therapeutics |
| 2022 | 14th HHT International Scientific Conference held in Portugal, with a record 282 experts | Community |
| 2022 | 40 Young Scholarship Travel grants awarded to rising clinicians and researchers | Research |
| 2022 | First virtual HHT Academy Patient and Physician conference hosted over nine days | Community |
| 2022 | New HHT Center of Excellence certified at the University of Florida | Care network |
| 2022 | $50,000 research grant awarded to Johns Hopkins for a standardized, validated quality of life metric for HHT | Research |
| 2021 | 30th anniversary of the foundation | Organization |
| 2021 | 30th HHT Center of Excellence in North America opened at Barrow Neurological Institute | Care network |
| 2021 | Guidelines webinar series delivered, 10 in English and six in Spanish | Guidelines |
| 2021 | International Expert Clinical Case series launched for the medical community | Community |
| 2021 | Department of Defense CDMRP funding received for a randomized trial of pazopanib in HHT-related bleeding | Therapeutics |
| 2021 | CDC public health webinar on HHT diagnosis and advances in treatment | Advocacy |
| 2020 | Second International Guidelines for the Diagnosis and Management of HHT published in the Annals of Internal Medicine | Guidelines |
| 2020 | Selected as one of only 30 organizations in the Chan Zuckerberg Initiative Rare As One Network | Organization |
| 2020 | Cure HHT Research Network launched, a patient-led research network | Research |
| 2020 | Redesigned Cure HHT Resource Library launched | Community |
| 2020 | First worldwide virtual walk, Strides for Strength | Community |
| 2020 | First HHT awareness billboard displayed during June Awareness Month | Community |
| 2019 | Christopher McMahon Memorial International HHT Guidelines Conference facilitated in Toronto, producing 36 recommendations | Guidelines |
| 2019 | Two new Centers of Excellence opened, at Winnipeg and Stanford Medical Center | Care network |
| 2019 | 13th International HHT Conference held in Puerto Rico, with attendees from 21 countries | Community |
| 2018 | Major HHT coverage in The New York Times and The Washington Post | Community |
| 2018 | My HHT Tracker released, the first free HHT iOS app | Community |
| 2018 | New Cure HHT website launched with a comprehensive resource library | Community |
| 2017 | Department of Defense awarded $10 million to five HHT researchers in a program project grant | Advocacy |
| 2017 | Federal funding secured for an HHT pilot initiative with three Hemophilia Treatment Centers in underserved areas | Advocacy |
| 2017 | 12th International Scientific and Research Conference held in Dubrovnik, Croatia | Community |
| 2017 | FDA engagement begun to establish a pipeline for therapeutic drug treatment of HHT | Therapeutics |
| 2017 | Protocols and standards updated for new and existing HHT Centers of Excellence | Care network |
| 2016 | North American Therapeutics group created to prioritize multi-center clinical and translational research | Therapeutics |
| 2016 | Expanded to 25 HHT Centers of Excellence in North America | Care network |
| 2016 | 25th anniversary of the foundation | Organization |
| 2015 | Young Scholar Research Program launched, investing $150,000 in young scholars | Research |
| 2015 | 11th Scientific and Research Conference held in Captiva, Florida, hosting 185 researchers | Community |
| 2014 | Renamed Cure HHT to reflect progress and aspirations | Organization |
| 2014 | Defense Appropriations Bill passed adding HHT to eligible research topics, opening access to over $247 million in federal grants | Advocacy |
| 2014 | HHT Physician Directory launched | Care network |
| 2014 | 20th HHT Center of Excellence established in North America | Care network |
| 2014 | Nosebleed study supported in collaboration with Stanford University | Research |
| 2013 | Expanded to 19 HHT Centers of Excellence in North America | Care network |
| 2013 | First North American Outcomes Registry funded, $131,000 | Research |
| 2012 | First Cure HHT Scientific Research Director appointed, Dr Marie Faughnan | Organization |
| 2011 | First professional HHT awareness video produced | Community |
| 2011 | Continuing medical education courses delivered to physicians and healthcare professionals | Community |
| 2011 | 9th International HHT Scientific and Research Conference supported, in Antalya, Turkey | Community |
| 2010 | First non-surgical multi-center clinical research study on nosebleed treatments launched | Research |
| 2010 | Expanded to 14 HHT Centers of Excellence in North America | Care network |
| 2010 | HHT Diagnosis and Treatment Act introduced in the House and Senate | Advocacy |
| 2009 | HHT Brain Vascular Research Consortia established with NIH funding | Research |
| 2009 | HHT Resolutions passed in the United States Congress, declaring June National HHT Awareness Month | Advocacy |
| 2008 | First Cure HHT Medical Director appointed, Dr James Gossage | Organization |
| 2008 | Four HHT research grants funded, totaling $170,000 | Research |
| 2008 | Commitment made to add two new Centers of Excellence every year | Care network |
| 2008 | First CDC-sponsored HHT conference held, on prevalence, early identification, and guideline adoption | Advocacy |
| 2007 | First nosebleed patient assessment tool funded, the Epistaxis Severity Score | Research |
| 2007 | Eleven HHT Centers of Excellence in North America | Care network |
| 2007 | First Capitol Hill Day hosted in Washington DC | Advocacy |
| 2006 | First HHT Clinical Guidelines Conference sponsored, producing international consensus on diagnosis, screening, and treatment | Guidelines |
| 2006 | Four HHT research grants funded, totaling $200,000 | Research |
| 2006 | NIH conference co-sponsored to identify priorities for HHT research | Research |
| 2004 | Dr Robert I White, Jr Young Clinician Award established | Research |
| 2004 | Expanded to nine HHT Centers of Excellence in North America | Care network |
| 2004 | First HHT research funding awarded, $55,000 for basic and clinical research | Research |
| 2003 | Release of the HHT gene patents obtained, making genetic testing possible for all patients in North America | Advocacy |
| 2003 | Three genetic testing laboratories funded in North America | Research |
| 2003 | First HHT capital research funding campaign launched | Organization |
| 2003 | HHT Research Grant Program established | Research |
| 1996 | Three HHT Centers of Excellence in North America | Care network |
| 1996 | HHT article published in the New England Journal of Medicine | Research |
| 1992 | First Patient and Family Conference held in Columbus, Ohio | Community |
| 1992 | First Scientific and Medical Advisory Board established | Organization |
| 1991 | HHT Foundation International established by Dr Robert I White, Dr Bruce Jacobson, and a small group of patients | Organization |
| 1991 | First HHT Center of Excellence established at Yale University School of Medicine | Care network |
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Who decides
The people behind the record
None of the entries above were decided by one person. Three groups set the direction, and every one of them is named in full on the About page.
40 members · 14 countries
Global Research and Medical Advisory Board
Clinicians and scientists across hematology, pulmonology, interventional radiology, genetics, pediatrics, and basic science. They guide the research agenda and the standards behind the Centers of Excellence.
Governance
Board of Trustees
Patients, family members, scientists, and business leaders who hold the organization to its mission and its finances. Several of them live with HHT themselves.
Day to day
Foundation staff and advisors
A small team running the research grants, the Centers of Excellence network, advocacy, education, and community programs, supported by senior clinical and scientific advisors.
What has not changed yet
Four out of five people with HHT still do not know they have it. The average family still waits decades for an answer. Severe nosebleeds still shape daily life for many people, and there is still no cure.
Listing what has been accomplished is only useful alongside what has not. The numbers to the right are the ones we measure ourselves against, and the ones we intend to change.
80%
still undiagnosed
27 years
average wait for a diagnosis
10–15%
of families with no identified genetic variant
0
cures, so far