A Foundation Built on Education and Determination

Dr. Zoghbi was born in Lebanon to parents who were passionate about education–her house was always stacked with books, schoolwork and learning were prioritized before chores or other household tasks, and all of her siblings went on to have successful careers in science, academia, business, and law. Although she originally wanted to pursue a degree in literature at the American University of Beirut, her mother was concerned about Zogbhi’s independence and convinced her to go into medicine.

During her first year of medical school, civil war broke out in Lebanon. With the country in turmoil, she and her classmates sought refuge underground at their school because it was often too dangerous to leave. Zoghbi spent the remainder of the semester sleeping in a sleeping bag on the floor of a women’s restroom because there was nowhere else for her to go.

In 1976, she and her younger brothers relocated to the United States to live with their older sister in Austin, Texas, where she continued her education at Meharry Medical College.

Discovering a Passion for Neurology

In 1980, Dr. Zoghbi moved to Houston for a pediatric residency at Baylor College of Medicine. Through her rotations, she developed a deep passion for neurology, ultimately becoming a child neurologist at Texas Children’s Hospital.

One patient in particular left a lasting impact on her—Ashley, a two-year-old diagnosed with Rett Syndrome, a newly discovered rare neurological and developmental disorder. When Dr. Zoghbi discovered several other young girls with similar symptoms who had been misdiagnosed, she knew she had to take action. Her deep empathy for these children and their families ignited a lifelong mission to uncover the genetic cause of the disorder.

Researching the Rett Syndrome Gene

Just five years into her medical career—and the same year she became a U.S. citizen—Dr. Zoghbi embarked on a postdoctoral fellowship in molecular genetics under the mentorship of Dr. Art Beaudet. Despite limited resources and years of setbacks, her instincts told her that Rett Syndrome had a genetic origin.

Nearly two decades after first encountering the disorder, she and her fellow researcher, Ruthie Amir, made a groundbreaking discovery in 1999: mutations in the MECP2 gene were responsible for Rett Syndrome. This discovery was not just a scientific breakthrough; it was a beacon of hope for affected families worldwide.

She credited her love of the girls she met who suffered from Rett Syndrome, along with her family and coworkers, for encouraging her to continue her research despite the constant roadblocks.

Dr. Huda Zoghbi’s Lasting Impact

Today, Dr. Zoghbi continues her vital research on the mechanisms of Rett Syndrome and other neurological disorders. She is the founder of the Jan and Dan Duncan Neurological Research Institute (NRI) at Texas Children’s Hospital and Baylor College of Medicine, where she fosters collaboration among young scientists tackling neurological diseases. Additionally, she is a professor at Baylor College of Medicine, an investigator at the Howard Hughes Medical Institute, and a recipient of numerous prestigious awards and grants.

Dr. Zoghbi’s journey reminds us that science is more than just data and experiments—it is fueled by curiosity, compassion, and the relentless drive to make the world a better place.

By celebrating trailblazers like Dr. Huda Zoghbi, we hope to inspire the next generation to dream big, push the boundaries of knowledge, and create lasting change through science.