Moyamoya Disease Research and Clinical Trials in Children

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Contact the Moyamoya Disease Program

At Boston Children’s Hospital, our care is informed by our research, and our discoveries in the laboratory strengthen the care we provide at each child's bedside. Boston Children’s scientific research program is one of the largest and most active of any pediatric hospital in the world. In particular, our neurology and neurosurgery researchers are yielding crucial insights into the causes and development of brain disorders, paving they way for the promising new treatments.

Learn more about ongoing research efforts in Boston Children's Neurobiology Program

Among our current research projects with promise for treating Moyamoya disease and pediatric stroke are: 

  • Evaluating possible growth factors in blood, cerebrospinal fluid
    Children’s neurosurgeon Edward Smith, MD, and his colleagues are studying molecular compounds and growth factors in blood and in cerebrospinal fluid. They believe that these growth factors may yield crucial insights into the causes and progression of Moyamoya disease, and may lead to new therapies not only for that disorder, but also for cancer and other conditions involving the proliferation of blood vessels.
  • Considering possible non-surgical treatments for Moyamoya
    Dr. Smith is also working to determine whether new drug therapies may provide a reasonable alternative to surgery for children with Moyamoya disease. Learn more about this work.
  • Identifying biological warning signs for Moyamoya disease
    Our researchers are working to identify telltale proteins in blood and urine that could serve as early “warning signals” for the presence of Moyamoya disease. Stay up to date on the team’s efforts.
  • Understanding the role of certain proteins in Moyamoya, other disorders
    Dr. Smith is investigating whether specific molecules may be culpable in the onset of several diseases of the central nervous system, including Moyamoya disease, brain tumors and vascular malformations of the brain.

Learn how you can support Moyamoya disease research at Boston Children’s.

Clinical trials 

Boston Children’s is known for pioneering some of the most effective diagnostic tools, therapies and preventive approaches in neurosurgery. A significant part of our success comes from our commitment to research—and to advancing the frontiers of pediatric health care by conducting clinical trials.

Boston Children’s coordinates hundreds of clinical trials at any given time. Clinical trials are studies that may involve:

  • evaluating the effectiveness of a new drug therapy
  • testing a new diagnostic procedure or device
  • examining a new treatment method for a particular condition
  • taking a closer look at the causes and progression of specific diseases 

Boston Children’s is involved in several multi-site clinical trials and studies focusing on pediatric neurology and neurosurgery, in particular. While children must meet strict criteria in order to be eligible for a clinical trial, your child may be eligible to take part in a study. Before considering this option, you should be sure to:

  • consult with your child’s treating physician and treatment team
  • gather as much information as possible about the specific course of action outlined in the trial
  • do your own research about the latest breakthroughs relating to your child’s condition 

Taking part in a clinical trial at Boston Children’s is entirely voluntary. Our team will be sure to fully address any questions you may have, and you may remove your child from the medical study at any time. 


We are grateful to have been ranked #1 on U.S. News & World Report's list of the best children's hospitals in the nation for the third year in a row, an honor we could not have achieved without the patients and families who inspire us to do our very best for them. Thanks to you, Boston Children's is a place where we can write the greatest children's stories ever told.”
- Sandra L. Fenwick, President and CEO

Boston Children's Hospital 300 Longwood Avenue, Boston, MA 02115 617-355-6000 | 800-355-7944

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