Neurofibromatosis Pediatric Research and Clinical Trials

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Contact the Multidisciplinary Neurofibromatosis Program

At Boston Children’s Hospital, we’re known for our science-driven approach and our intense culture of innovation—a philosophy that lets us push the boundaries of pediatric care.

We’re one of nine sites selected country-wide for expertise in taking care of patients with neurofibromatosis to participate in the Department of Defense-funded Clinical Trials Consortium. As part of the Consortium, we run trials aimed at finding new ways to treat:

  • progressive plexiform neurofibromas
  • pediatric brain tumors
  • pediatric learning disabilities
  • malignant peripheral nerve sheath tumors

We also lead and participate in clinical studies that investigate:

  • growth abnormalities in children with NF1, including premature puberty
  • bone density in neurofibromatosis
  • the relationship between a mutation and the symptoms that people with that mutation may or may not develop
  • early ways to detect complications of NF1
  • how new blood vessels are created in tumors associated with NF1

If you’d like to find out more about our research and clinical trials or see whether your child might be a candidate, please talk to your child’s doctor or email us at

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
For Patients: 617-355-6000
For Referring Providers: 844-BCH-PEDS | 844-224-7337