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Division of Neurology Research Studies
1 - 14 of 14
AxD Outcomes
A thorough review of outcome measures that assess disease progression has not been established for any of the subcategories of Alexander disease.
Related Conditions:
This study is actively recruiting.
Friedreich Ataxia Clinical Outcomes Measures
This is a multicenter natural history and clinical measure study.
Related Conditions:
This study is actively recruiting.
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.
Related Conditions:
This study is actively recruiting.
Natural History Study for SYNGAP1 Related Disorders
The objective of the study is to better define and outline the clinical spectrum of SYNGAP1 through detailed developmental, seizure, and quality of life assessments as an extension of routine clinical care.
Related Conditions:
This study is not recruiting.
Neurophysiological Biomarkers in Rett Syndrome
The purpose of this study to learn more about the brain function of females with Rett Syndrome and how it compares to the brain function of typically developing females.
Related Conditions:
This study is actively recruiting.
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.
Related Conditions:
This study is actively recruiting.
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.
Related Conditions:
This study is actively recruiting.
PERCEPTION Study
The PERCEPTION Study is a collaborative effort between several institutions in the US.
Related Conditions:
This study is not recruiting.
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.
Related Conditions:
This study is not recruiting.
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.
Related Conditions:
Leukodystrophies, Leukodystrophy, Biorepository, Pediatric Neurology, Neurological and Neuromuscular , Genetic Disease, Genetic Disorders, Rare Genetic Disorders, Pediatric Genetic Disease, Aicardi Goutieres Syndrome, TUBB4A, Metachromatic Leukodystrophy, Adrenoleukodystrophy, Leukoencephalopathies, Leukoencephalopathy, Biobank, Biobanking, H-ABC, Alexander Disease, Krabbe Disease, Whole Genome Sequencing, Exome Sequencing, Whole Exome Sequencing, Next Generation DNA/RNA Sequencing, Next-generation Sequencing, Rare Disorder Natural History Studies, Rare Disease
This study is actively recruiting.
The SLEEPY Study
The SLEEP study will test different sleep promoting strategies for children.
Related Conditions:
This study is not recruiting.
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.
Related Conditions:
This study is actively recruiting.
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.
Related Conditions:
This study is not recruiting.
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.
Related Conditions:
This study is actively recruiting.