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Division of Neurology Research Studies
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A thorough review of outcome measures that assess disease progression has not been established for any of the subcategories of Alexander disease.
This is a multicenter natural history and clinical measure study.
While recent research has demonstrated that whole genome sequencing is a powerful first-line diagnostic tool, important questions remain around its long-term impact on downstream clinical management approaches.
CHOP is conducting a research study that will help make it possible to do future studies in children and family members both with and without problems with their brain and nervous system.
The purpose of this study is to create a biorepository to support our ongoing and future studies on ultra-rare pediatric neurodegenerative disorders.
The PERCEPTION Study is a collaborative effort between several institutions in the US.
The aim of this study is to determine if NB-001 (an investigational drug that is not approved by the FDA) is effective, safe, and well tolerated in treating children and adolescents with 22q11DS and anxiety, inattention, and/or autism.
This study involves the collection of medical information and samples from people with a confirmed or suspected leukodystrophy or related disorder affecting the white matter of the brain, as well as "healthy controls" who are not known to be affected by one of these disorders.
The SLEEP study will test different sleep promoting strategies for children.
This study is a trial comparing the effects of erenumab to placebo in children ages 6-17 years old with migraine 15 days per month or more.