Pediatric Neurogenetics Disorders Program

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Pediatric Neurogenetics Disorders Program

Gene therapies and advanced treatments for neurogenetic disorders in children

Our doctors are national leaders in gene therapies and other innovative treatments. We pinpoint changes in genes that cause certain neurogenetic disorders and customize treatments to help your child move toward a happier, healthier future.

The comprehensive Neurogenetics Disorders Program at Children’s Health℠ features full genomic sequencing and the latest gene replacement therapies. We specialize in analyzing a child’s DNA to select the most effective treatment for their unique condition.

We also give your family access to more clinical trials than almost anywhere else in the country. Our doctors’ active involvement in national research and clinical trials means your family can have access to the latest, most promising therapies.

Expert genomic sequencing and gene therapies

Doctors in our Neurogenetic Disorders Program don’t just diagnose your child’s disorder – we look for its root cause. Our medical geneticists use a method called full genomic sequencing, which allows us to look at a child’s DNA in great detail. This information helps our doctors identify the specific gene or chromosome change that’s causing a child’s illness. And the more we know about the root cause of a genetic disease, the closer we get to finding a gene treatment and, someday, a cure.

We’re among a select few children’s hospitals with a dedicated focus on gene therapies for children. Gene therapies seek to replace missing or faulty genes with healthy ones. Once we understand your child’s condition, we can determine if there is a gene therapy option for them.

We offer access to a number of gene therapies that are currently available, and we’re actively engaged in research to develop new ones.

Developing new gene therapies for neurogenetic disorders

Our doctors are also faculty members at UT Southwestern, one of the country’s top biomedical research institutions. These physicians are leading a national program to create new gene replacement therapies to help children with a wide range of pediatric neurogenetic disorders. The aim is to find new ways to treat – and someday prevent – neurogenetic disorders in children.

As gene therapy evolves, more and more new therapies are becoming available via clinical trials. We offer access to more gene therapy clinical trials than almost any other center, and we partner with national foundations to help more families participate in these trials. We’re also actively involved in launching new clinical trials that could help children in our region and nationwide.

Comprehensive, top-rated care

Children’s Health is home to experts who work together to select the treatment that gives your child the best opportunity for a good outcome, no matter which neurogenetic disorder they have. Sometimes gene therapy is a good option. Other times, it might be better to consider other treatments and/or surgery. We have the expertise to find the treatment that’s right for your child, and we are regularly recognized for top neurosurgery and neurology care by U.S. News & World Report.