Texas Children's 2021 Annual Report

The Brain Prize recognizes Dr. Huda Zoghbi for her groundbreaking and life-changing research


Dr. Huda Zoghbi, director of the Jan and Dan Duncan Neurological Research Institute, accepted the Lundbeck Foundation Brain Prize, the largest prize for neuroscience in the world, on November 4, 2021. Sir Adrian Bird of the University of Edinburgh, Scotland, was the co-recipient of the award, which was presented during a ceremony in Copenhagen presided over by His Royal Highness Crown Prince Frederik of Denmark.


For more than three decades, Drs. Zoghbi’s and Bird’s cumulative and pioneering research has led to an increased understanding of Rett syndrome and has brought patient families hope that there might one day be a treatment for the disorder, a devastating neurodevelopmental condition primarily affecting girls.


Dr. Zoghbi received this award for her role in discovering a gene mutation that causes Rett syndrome and for establishing the importance of epigenetic regulation in both brain development and maintenance of normal adult brain function. These discoveries also point to novel opportunities for treatment of this and other neurodevelopmental disorders.


In 1983, Dr. Zoghbi began studying Rett syndrome and, more than 15 years later in 1999, she identified the gene mutation that causes the disorder. This monumental discovery created a path to developing a diagnostic genetic test, allowing early and accurate diagnosis of Rett syndrome. Her research revealed that Rett syndrome is caused by mutations in the MECP2 gene that encodes a protein with the same name, MeCP2, which Sir Adrian Bird identified in 1992.



Formerly known as the Grete Lundbeck European Brain Research Prize, the annual award recognizes highly original and influential advances in any area of brain research. The prize, worth over $1.5 million, is awarded to one or more scientists who have distinguished themselves through outstanding contributions in their field. Brain Prize winners are decided by a panel of the world’s top neurological researchers after nomination by peers.


“I am deeply honored and humbled to receive the Brain Prize,” said Dr. Zoghbi, who is also a Howard Hughes Medical Institute Investigator and Professor at Baylor College of Medicine. “I share this with all my trainees, past and present, who have worked so hard to advance research on Rett syndrome. I am grateful to the Lundbeck Foundation for this prize and what it means for our ability to continue pushing the boundaries of discovery to help people with Rett syndrome and MECP2 duplication disorder.”


“When we embarked on the journey of opening the Jan and Dan Duncan Neurological Research Institute, I knew Dr. Zoghbi and her team would change millions of lives and ultimately change the world,” said Texas Children’s Hospital President and CEO Mark A. Wallace. “Dr. Zoghbi is one of the most dedicated and passionate neuroscientists I’ve ever met. Her unmatched commitment to further understanding the causes of neurological and neurodegenerative diseases is only one of the many reasons she deserves this prestigious award.”



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