- Conditions
- Epilepsy, Seizure, Cognition Disorders
- Interventions
- Differential Abilities Scale, Neuropsychological Testing
- Behavioral
- Lead sponsor
- Emory University
- Other
- Eligibility
- Female only
- Enrollment
- 331 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2000 – 2012
- U.S. locations
- 20
- States / cities
- Birmingham, Alabama • Tucson, Arizona • Los Angeles, California + 17 more
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- Conditions
- Lysosomal Diseases, Gangliosidosis, GM1
- Interventions
- AAV9-GLB1, Abdominal ultrasound, Rituximab, Sirolimus, Methylprednisolone, Prednisone, Audiology assessment with ABR, Bone density scan (DEXA), Electrocardiogram (EKG), Echocardiogram, Electroencephalogram (EEG) awake and extended overnight, Laboratory tests, Lumbar puncture, Brain MRI/MRS/fMRI, Neurocognitive testing, Neurology exam, PICC or other Central line placement, Skeletal survey, Skin biopsy, Speech and modified barium swallow study, Ophthalmology exam
- Biological · Procedure · Drug + 3 more
- Lead sponsor
- National Human Genome Research Institute (NHGRI)
- NIH
- Eligibility
- 6 Months to 12 Years
- Enrollment
- 54 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2028
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Infant Development
- Interventions
- Bayley Scales of Infant Development-II, Bayley Scales of Infant Development-III
- Diagnostic Test
- Lead sponsor
- Children's Hospital of Philadelphia
- Other
- Eligibility
- 18 Months to 22 Months
- Enrollment
- 119 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2011 – 2015
- U.S. locations
- 1
- States / cities
- Philadelphia, Pennsylvania