Last updated on February 2018

Longitudinal Study of Neurodegenerative Disorders


Brief description of study

The purpose of this study is to understand the course of rare genetic disorders that affect the brain. This data is being analyzed to gain a better understanding of the progression of the rare neurodegenerative disorders and the effects of interventions.

Detailed Study Description

Patients would be evaluated by a multidisciplinary team at specific time points every 3 months the first year, every 6 months the second year and once a year thereafter.

Clinical Study Identifier: NCT03333200

Contact Investigators or Research Sites near you

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Maria L Escolar, MD,MS

The Program for the Study of Neurodevelopment in Rare Disorders, Children's Hospital of Pittsburgh of UPMC
Pittsburgh, PA United States
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