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Huda Y. Zoghbi, M.D.

Huda Y. Zoghbi, M.D. is an internationally renowned neurogeneticist and the founding director of the Jan and Dan Duncan Neurological Research Institute (NRI) at Texas Children’s Hospital. She is also an Investigator with the Howard Hughes Medical Institute and a professor of Pediatrics, Neurology, Neuroscience and Molecular and Human Genetics at Baylor College of Medicine (BCM). In addition, Dr. Zoghbi is a member of the National Academy of Medicine, the National Academy of Sciences, and the American Academy of Arts and Sciences. She is also a trustee of the American University of Beirut, Rice University, and The Rockefeller University.

Born and raised in Lebanon, Dr. Zoghbi received her undergraduate degree in biology from the American University of Beirut and began medical school there in 1975.  The civil war in Lebanon broke out during her first year of studies. After she spent a semester living in the basement of the university to stay safe, Dr. Zoghbi moved to the United States, where she transferred to Nashville’s Meharry Medical College. 

Dr. Zoghbi completed a pediatric residency and a residency in pediatric neurology at BCM in 1985. Seeing children with incurable, inherited neurological diseases and not being able to provide them with hope piqued her interest in research and led her to pursue a postdoctoral fellowship in molecular genetics under the mentorship of the preeminent geneticist Dr. Arthur Beaudet.  She joined the Baylor’s faculty as an assistant professor in 1988. Dr. Zoghbi’s research spans neurodevelopment to neurodegeneration, and has led to key discoveries in Spinocerebellar Ataxia type 1, Rett syndrome, Bipolar disorder and Alzheimer’s disease. She has authored over 300 peer-reviewed publications in top-tier journals.

In 2010, Dr. Zoghbi founded the Duncan NRI at Texas Children’s Hospital to develop treatments for brain disorders, which impact one billion people, including 300 million children. Under her leadership, the NRI has grown to over 30 principal investigators and 300 research trainees with a track record of groundbreaking discoveries, including:

  • 60+ genetic mutations that underlie neurological diseases—10% of all those known worldwide. 
  • A master gene that holds the key not only to a rare pediatric neurological disorder, but also to adult-onset diseases like Parkinson’s.
  • The use of an established cancer drug to treat forms of severe childhood epilepsy.
  • A neural circuit in the brain that controls addiction and eating disorders.

Awards and Honors

In addition to the Brain prize, Dr. Zoghbi has received several prestigious international awards and honors. Some of her recent accolades include:

The Breakthrough Prize in Life Sciences 

For discoveries of the genetic causes and biochemical mechanisms of spinocerebellar ataxia and Rett syndrome, findings that have provided insight into the pathogenesis of neurodegenerative and neurological diseases.

The Canada Gairdner International Award 

For the discovery of the genetic basis of Rett syndrome and its implications for autism spectrum disorders.

The Shaw Prize in Life Science and Medicine 

For fundamental discoveries in biomedical science and innovations in clinical medicine that have led to significant victories in our longstanding war against illness and suffering.

The March of Dimes Prize in Developmental Biology 

To honor investigators whose research advances the science that underlies birth defects.

American Society of Human Genetics’ Victor A. McKusick Leadership Award

This award, named in honor of the late Victor A. McKusick, MD, recognizes individuals whose professional achievements have fostered and enriched the development of human genetics as well as its assimilation into the broader context of science, medicine, and health. 

National Order of the Cedar, Knight grade

Dr. Zoghbi also holds honorary degrees from Yale University, Harvard University and the University of Massachusetts Medical School. She and her husband, Dr. William Zoghbi, have two children—Anthony and Roula—and three grandchildren.