Care for Children with Leukodystrophies

The Leukodystrophy Center at Children's Hospital of Philadelphia provides comprehensive care for children with leukodystrophies. www.chop.edu/leukodystrophy
Leukodystrophies are inherited disorders of the white matter of the brain and spinal cord. The white matter is made up of specialized cells and a substance called myelin. Myelin is like the insulation around the electrical cords of your brain that allow you to speak, walk, eat and breathe. Children and adults affected by leukodystrophies can have problems in all those areas.
Leukodystrophies are difficult to recognize and diagnose. A leukodystrophy diagnosis raises many other questions. The Leukodystrophy Center at Children's Hospital of Philadelphia (CHOP) provides comprehensive clinical care, diagnostic testing and the most advanced leukodystrophy treatments available to infants, children and adolescents living with these inherited white matter diseases.
In this video, hear from families who have been cared for by CHOP’s Leukodystrophy Center and the physicians who are leading the team, including Adeline Vanderver, MD, Amy Waldman, MD, and Brenda Banwell, MD.
The Leukodystrophy Center includes a newborn screening program that identifies infants who have leukodystrophy. A multidisciplinary, comprehensive care clinic focuses on helping address the needs of patients living with leukodystrophy, including diagnosing the disorder and managing the various leukodystrophy symptoms to maximize health, quality of life and function.
The therapeutic arm of the program focuses on bringing advances to clinical treatment by offering current clinical trials as well as the next generation of clinical trials when they’re available. A discovery program uses cutting-edge leukodystrophy research to better understand the causes and biology of leukodystrophy with the goal of translating that research into meaningful advances in clinical care.
Overall, the Leukodystrophy Center team focuses on bringing together clinicians, families and patient advocacy groups to change what it means to live with inherited white matter disorders.