Henry’s Parents Visit the Duncan NRI

April 2023

On April 11, Mary Forrest and Richard Engel, Henry’s parents, traveled to Houston to meet with researchers – including Dr. Huda Zoghbi – who are actively working on a cure for Rett syndrome.

Given Richard and Mary’s tremendous support of this research, they are pictured here on the newly named ‘Henry Engel Memorial Balcony’ receiving photos of Henry’s cells that are the building blocks for discoveries that can one day save children’s lives.

What is Rett syndrome?

Rett syndrome is a rare genetic neurological disorder that affects the way the brain functions. This disorder primarily affects females, causing a progressive loss of motor skills and language. Worldwide, it is estimated to affect about 1 in 10,000 girls. Numerous disabilities, ranging from mild to severe, are typical in Rett syndrome. Parents may first notice symptoms around 6-18 months when their child misses key developmental milestones. Eventually, it leads to severe impairments that affect a child’s ability to speak, walk, eat and breathe easily. A hallmark of Rett syndrome is near-constant repetitive hand movements (hand wringing). Most affected individuals also experience seizures at some point. Some patients suffer from irregular heartbeats, sleep disturbances and scoliosis (sideways curvature of the spine). 

A vast majority of cases are caused by spontaneous mutations in the X-linked gene called methyl-CpG-binding 2 (MECP2); very few are caused by familial inheritance. Most girls with MECP2 mutations develop normally and then start showing signs of regression. Females have two copies of the MECP2 gene (one in each of their X-chromosomes), which means half of their cells have a healthy copy of the gene and half have a mutant copy. However, since males do not have a second set of X chromosomes, every cell will have the mutant gene and so the consequences of MECP2 mutations are much more severe — most die during infancy or early childhood.

Currently there is no cure for Rett syndrome and most treatments are palliative.

Latest treatment advances for Rett syndrome

Dr. Huda Zoghbi, founding director of the Duncan NRI at Texas Children’s Hospital and Professor at Baylor College of Medicine, is widely renowned for her groundbreaking research on Rett syndrome and the MECP2 gene and for developing a strategy to normalize MeCP2 levels, with studies showing that this reverses disease symptoms in a mouse model of MECP2 duplication syndrome. This is an exciting milestone for patients, families and caregivers — a first step on a long road toward a cure. Her team is currently focused on finding therapeutic strategies that can help people with Rett syndrome.