Canavan Disease Clinical Trial
Official title:
Oral Glyceryl Triacetate (GTA) in Newborns With Canavan
Canavan disease is caused by Aspartoacylase deficiency. There is no treatment for the disease, but there is a food additive that includes acetate . We suggest an early treatment with acetate and a neurologic evaluation, including MRI, after 4 months of treatment. In any case the treatment will be stopped at the age of 22 months, when myelinization is ended.
n/a
Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
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