Gorham-Stout Disease Pediatric Research and Clinical Trials

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Contact the Vascular Anomalies Center

At Boston Children’s Hospital, our team is world renowned for its expertise in Gorham-Stout disease because we do more than just treat it. Our experts are actively involved in the latest research on this disease, including participation in leading clinical trials.

Our dedication goes beyond treating today’s patients. Our goal is to find more effective treatments to halt the disease or even cure it.

Gorham-Stout Research at Boston Children’s

Clinical Research

Our patients have access to the latest clinical protocols for investigational drug therapies. We are currently studying the use of immunosuppressant medications to slow and even halt the progression of Gorham-Stout disease.

Results have already shown significant success in patients, with the disease stabilizing or reversing course in most participants. Read more about our Gorham-Stout disease treatment.

We are also very active in the collection of patient data related to Gorham-Stout disease, with the world’s largest comprehensive registry of patients.  Through this registry, we are studying causes of mortality, effusions, infections and other challenges in Gorham-Stout disease.  For more information or to participate, please visit our Lymphatic Anomalies Registry.

Translational Research

In addition to clinical research, our team is at the forefront of translational research into Gorham-Stout, including studies looking at possible genetic causes. The goal is to translate discoveries in the lab into medical treatments to help patients.

Research is currently underway to determine whether a genetic mutation could be the cause of this rare bone disorder. There is currently no known cause of Gorham-Stout.

Reviewed by Cameron Trenor, MD, © Boston Children’s Hospital

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- Sandra L. Fenwick, President and CEO

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