The Gilbert Family Neurofibromatosis Institute

The goal of the Gilbert Family Neurofibromatosis Institute at Children’s National Hospital is to help children with neurofibromatosis type 1 or 2 (NF1, NF2) live more normal lives. It is one of the world’s largest programs, and the longest standing program in the United States, changing the lives of children and families since 1982. This experience ensures that your child will have access to unparalleled expertise and care.         

Helping Children With Neurofibromatosis

Our dedication to your child’s health and well-being is why we’ve brought together some of the best and brightest minds on our medical and research teams. 

Highlights of our program include:

• Expertise. Our clinical/translational team is led by Roger J. Packer, M.D., an internationally-recognized pediatric neurologist who brings his unique depth of experience in innovative clinical research, diagnosis and treatment and Miriam Bornhorst, M.D., a pediatric neuro-oncologist dedicated to the field of NF.
• Team approach. From neurologists to eye experts (neuro-ophthalmologists) to geneticists to oncologists, our world-class sub-specialist team is experienced in meeting the complete, unique needs of each neurofibromatosis patient.
• Seamless care. We offer several multidisciplinary clinics, giving your child access to a broad range of specialists in one central location. Learn more about our related departments.
• Support. Having a child with neurofibromatosis can affect your entire family, and we are here to help. We offer services to meet your family’s emotional, spiritual and mental health needs. We were also one of the first programs in the world to offer a summer camp just for children with neurofibromatosis. Camp New Friends is the largest neurofibromatosis summer camp on the East Coast.

World-Class Neurofibromatosis Care at Children’s National

We offer highly specialized treatments for your child, some of which are only available at Children’s National. In fact, we have successfully treated conditions and complications that were previously thought to be untreatable.

Using a personalized approach, we coordinate treatments, therapies and support to relieve symptoms while protecting your child from long-term complications.

Highlights of the world-class neurofibromatosis care and programs at Children’s National include:

• Neurologic, neuro-oncologic and oncology care. Our clinical team led by Drs. Bornhorst, Packer and Aerang Kim, M.D., (pediatric oncologist, Malignant Peripheral Nervous System Tumor Program director) provides unparalleled care and develops and leads national and international studies providing innovative, life-changing care.
• Life-changing therapeutic studies. The Gilbert Family Neurofibromatosis Program, a founding member of the Department of Defense Neurofibromatosis Clinical Trials Consortium and other national consortium (Sarcoma Alliance, Pediatric Brain Tumor Consortium) offers therapeutic options available in few programs around the world.

• Ophthalmologic expertise. Steven Stasheff, M.D., within our Division of Neuro-Ophthalmology, specializes in caring for complications affecting your child’s vision with a special emphasis on neurologic complications and visual pathway gliomas. We offer treatments that can improve or save your child’s eyesight.
• Cognitive rehabilitation. Kristina Hardy, Ph.D., and Karin Walsh, Psy.D., lead pioneering efforts to improve working memory and brain functioning through cognitive rehabilitation. This therapy uses computer exercises to strengthen specific brain activities.
• Genetic expertise and care. Led by Kenneth Rosembaum, M.D. (pediatric geneticist) and Dr. Bornhorst (Oncologic Neurogenetics leader) the program has a long tradition of integration of genetics and genetic counseling into NF1 and NF2 care.
• Laboratory/translational research. In addition to our unmatched clinical trials experience, which gives your child access to new medications, including targeted therapies, and soon immunologic therapies, our laboratory-based research program under the direction of Yuan Zhu, Ph.D., (scientific director of the Gilbert Family Neurofibromatosis Institute) and now with the addition of Javad Nazarian, Ph.D., M.Sc., the program is performing laboratory research which focuses on NF1-related low-grade glioma, transformed gliomas, plexiform neurofibromas and malignant peripheral nerve sheath tumors to develop new therapeutic approaches for these NF1 complications. Ours is one of the largest NF1-dedicated research programs in the world and provides its clinicians new avenues to explore to improve outcomes.
• Family of other specialists. Given the complexity of the needs of children and families with NF1 and NF2, in addition to the daily involvement of neurology, genetics, oncology, neuro-ophthalmology and neuropsychology, there is active involvement in the multidisciplinary clinic by developmental pediatrics (Dr. Uy), orthopedics (Dr. Oetgen), Physical Medicine and Rehabilitation (Dr. Shin), psychiatry, psychology, ENT, endocrinology, plastic surgery, neurosurgery and hearing and speech.
• Adult care. Physicians board-certified in both pediatric and adult neurology ensure a smooth transition to adult care. If your child’s case is complex, he or she can stay at Children’s National. We are one of few pediatric programs in the country capable of caring for adults.

Conditions We Treat

Our team treats conditions related to neurofibromatosis, including:

• Optic nerve gliomas and other low-grade gliomas
• Transformed anaplastic gliomas
• Plexiform neurofibromas
• Malignant peripheral nerve sheath tumors
• Learning disabilities and behavioral challenges
• Autism spectrum disorders associated with NF1
• Acoustic schwannomas and associated hearing/language deficits
• Other pediatric neurofibromatosis-related tumors
• Orthopedic complications including scoliosis and tibial dysplasia
• Meningiomas
• NF1-related hormonal dysfunction
• Intraspinal ependymomas

International Leaders in Neurofibromatosis Research

Our experts share a commitment to developing safer, more effective therapies that help reverse the course of neurofibromatosis, including NF1 and NF2. Through clinical trials, your child has access to new treatments that are only available in select programs worldwide. 

Another aspect of our NF1 and NF2 program which distinguishes Children’s National is our leadership in some of the world’s most prestigious neurofibromatosis research networks.

Notable accomplishments include:

• Chairing the only neurofibromatosis research consortium in the world: the Department of Defense-funded Neurofibromatosis Clinical Trials Consortium
• Becoming one of the leading programs in the Sarcoma Alliance for Research through Collaboration (SARC Alliance) for developing new molecularly-targeted therapies
• Being recognized by the Children's Tumor Foundation as a Neurofibromatosis Center of Excellence

Contact Us

For more information or to make an appointment, call us at 202-476-5181.