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Division of Neurology Research Studies
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AxD Outcomes

A thorough review of outcome measures that assess disease progression has not been established for any of the subcategories of Alexander disease.
Friedreich Ataxia Clinical Outcomes Measures

This is a multicenter natural history and clinical measure study.
LeukoSEQ: First-Line Whole Genome Sequencing in Leukodystrophies

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.
Neurophysiological Biomarkers in Rett Syndrome
Neuroscience Center Biorepository

The goal of this study is to collect biological samples and medical information from children to create a Neuroscience Biorepository to advance future neurological research.
Pediatric Neurodegenerative Biorepository
The purpose of this study is to create a biorepository to support our ongoing and future studies on ultra-rare pediatric neurodegenerative disorders.
PERCEPTION Study
The PERCEPTION Study is a collaborative effort between several institutions in the US.
Safety and Efficacy of NB-001 in 22q11DS Children with Anxiety, Inattention, and/or Autism

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.
The Myelin Disorders Biorepository Project (MDBP)

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 SLEEPY Study

The SLEEP study will test different sleep promoting strategies for children.
ThecaFlex Investigational Device for the Treatment of SMA

Spinal muscular atrophy (SMA) is a neuromuscular disease characterized by the degeneration of spinal cord motor neurons, resulting in progressive muscle weakness, paralysis, and atrophy.
Trial of Erenumab for Preventive Treatment of Pediatric migraine 15 days per month or more

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.
Trial of Erenumab in Children and Teens with Migraine less than 15 days per month

This study is a trial comparing the effects of erenumab to placebo in children ages 6-17 years old with migraine less than 15 days per month.