Dietary Supplements for the Treatment of Angelman Syndrome
Angelman Syndrome, Nervous System Diseases
About this trial
This is an interventional treatment trial for Angelman Syndrome focused on measuring Developmental Delay, Mental Retardation, Ataxia, Microcephaly, Seizures
Eligibility Criteria
Inclusion Criteria: Diagnosis of AS In stable condition with relatively good control of seizures Willing to comply with treatment, study visit schedule, and study assessments Willing to take oral or G-tube medication Willing to be contacted monthly during the course of the study Parent or guardian willing to provide informed consent Exclusion Criteria: History of liver or kidney disease Currently being treated for a serious acute illness Known hypersensitivity to any of the study drugs Received high-dose folate drug treatment in the 12 months prior to study entry Other significant medical problems, including those involving the liver, kidney, or heart Other comorbidities, genetic disorders, or extreme prematurity; children with autism are not excluded
Sites / Locations
- Rady Children's Hospital San Diego
- Children's Hospital Boston
- Greenwood Genetics Center
- Baylor College of Medicine
Arms of the Study
Arm 1
Experimental
1
Participants will receive two daily doses of Metafolin, betaine, and creatine, and one daily dose of vitamin B12 for 12 months.