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Drug Therapy

Drugs are approved in the U.S. by the Food and Drug Administration (FDA) for specific purposes to help guide medical practice.

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Currently, there are no FDA approved drugs specifically for the treatment of HHT. However, there have been several promising studies and positive results for medications originally developed to treat other conditions.

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Related Resources

Entering the Therapeutics Age: A New Era for HHT Treatment (Recorded)

Webinar – 2024

Lately, there’s been an exciting wave of news about HHT therapies. Several clinical trials are now underway, biotech companies are investing heavily in HHT research, and new treatments are closer than ever to becoming reality. We’re entering this new…

How to Be an HHT Changemaker Part 1: Drive Research (Recorded)

Webinar – 2024

Participating in research is the ultimate way to be an HHT changemaker. We have two exciting new projects to share with you, HHT Connect and the Cure HHT BioBank. Courtney Kasturiarachi, MPH, walks you through everything you need to know about HHT Co…

NEW Criteria! Groundbreaking Trial for HHT: Learn About Pazopanib (Recorded)

Webinar – 2024

Your questions answered about our newest clinical trial with NEW expanded criteria! Join Cassi Friday, PhD and Jim Gossage, MD as they explain who is now eligible, what Pazopanib is, and how you can get involved!

Caring for HHT Through the Generations (Recorded)

Webinar – 2024

Drs. Adrienne Hammill, Center Director at Cincinnati Children’s Hospital and Kevin Whitehead, Center Director at University of Utah. Dr. Hammill guides you through the early stages of life, covering genetic testing, prenatal care, and the significanc…

Pazopanib: A Groundbreaking Trial for HHT (Recording)

Webinar – 2023

Get your questions answered about our newest clinical trial! Join Cassi Friday, PhD and Jim Gossage, MD as they explain who is eligible, what Pazopanib is and how you can get involved!

Bedside to Bench: How the HHT Community Drives Research Initiatives (Recorded)

Webinar – 2021

In March 2020, Cure HHT was 1 of 30 organizations selected to participate in the Chan Zuckerberg Initiative (also known as CZI) Rare is One Project. This ambitious project is lifting up the work that patient communities are doing to accelerate resear…

Nuts and Bolts of A Clinical Trial – the PATH-HHT Study (Recorded)

Webinar – 2021

Research is the key to the future health and quality of life for HHT patients. “Diseases can only be cured when scientists understand them. This understanding, which comes from basic research, leads to new drugs and treatments.” ~Dr. Christopher Hugh…

International HHT Guidelines – Overview (Recorded)

Webinar – 2021

Join Dr. Marie Faughnan, Director, Toronto HHT Centre of Excellence as she provides an overview of the newly updated International HHT Guidelines and how they are used to set the standard of care globally for the diagnosis and treatment of HHT. Dr. F…

Bevacizumab (Avastin) Use In HHT

Policy Statement – 2020

Hereditary hemorrhagic telangiectasia (HHT, also known as OslerWeber-Rendu Syndrome) is a condition that is characterized by focal proliferation of blood vessels (vascular malformations) in multiple organs. Two of the most commonly affected organs in…

Bevacizumab for treating HHT patients with severe hepatic involvement or refractory anemia

Scientific Literature – 2020

PLoS One. Authors: Carolina Vázquez, María Laura Gonzalez, Augusto Ferraris, Juan Carlos Bandi, and Marcelo Martín Serra. This article is available for public use and does not require purchase. Click to view the full article.

The Science Behind the 2019 Scientific Conference (recorded)

Webinar – 2020

Philippe Marambaud, PhD and Andy White, MD discuss important advances in HHT research which were presented at the 2019 HHT International Scientific Conference in Puerto Rico. Learn about advancements in drug therapies for bleeding, screening and trea…

Systemic bevacizumab for high-output cardiac failure in HHT: an international survey of HHT centers

Scientific Literature – 2019

Orphanet Journal of Rare Diseases. Authors: Hanny Al-Samkari, Hasan A. Albitar, Scott E. Olitsky, Marianne S. Clancy and Vivek N. Iyer. This article is available for public use and does not require purchase. Click to view the full article.

Systemic bevacizumab for the treatment of chronic bleeding in HHT

Scientific Literature – 2018

Journal of Internal Medicine. Authors: Al-Samkari, H., Kritharis, A., Rodriguez-Lopez, J.M., and Kuter, D.J. This article can be purchased directly from the publisher. Click to view the article description and purchase options.

HHT: Diagnosis and Management from the Hematologist’s Perspective

Scientific Literature – 2018

Haematologica. Authors: Athena Kritharis, Hanny Al-Samkari, and David J Kuter. This article is available for public use and does not require purchase. Click to view the full article.