Center for the Treatment of Pediatric Neurodegenerative Diseases

Coordinated Clinical Care

The Center for the Treatment of Pediatric Neurodegenerative Disease strives to centralize care for children afflicted with these rare, chronic, and debilitating diseases. The Center includes a multidisciplinary team of physicians and other healthcare providers offering comprehensive services to aid in the diagnosis, management, and social support of children and their families suffering from neurodegenerative diseases. In coordination with each patient, their family, and their primary care physician, the team develops a personalized long-term management plan that is uniquely tailored to each patient’s needs.

The Center is comprised of multiple sub-specialty clinics such as the Mitochondrial Clinic, the Leigh Syndrome Clinic, the Lysosomal Clinic, the Ataxia Telangiectasia Clinic, and Pedi Move. Disorders and diseases falling within these sub-specialty clinics are multisystem disorders requiring coordinated care through multiple physicians and subspecialists. With the creation of this Center, children suffering from these rare, chronic and debilitating diseases are now able to receive care in one centralized location.

More on the prevalence of neurodegenerative disease

It is estimated that 1 in 4 Americans will develop a neurodegenerative disease. These conditions are incurable and debilitating, resulting in the progressive degeneration — or death — of nerves. More than 600 disorders, most of which are rare, can be included in this category. The neurodegenerative diseases of infancy and childhood represent a particularly devastating subset of these conditions in which previously healthy children begin to lose control of their nervous system. Unlike other neurologic diseases of childhood, neurodegenerative diseases escalate quickly, are often misdiagnosed, and typically lead to an early death. While most neurodegenerative diseases of childhood are caused by genetic mutations, the underlying mechanics of neurodegeneration is not fully understood. Improved understanding about these conditions will lead to enhanced therapies and better methods for decreasing morbidity and improving survival in these complex patients.