Efficacy of Minocycline for the Treatment of Angelman Syndrome | $180,000.00

Angelman syndrome is a rare genetic disorder characterized by phenotypic traits such as global developmental delay, ataxia and seizure. Children diagnosed with AS display a behavioral profile consisting of a happy demeanor with easily provoked laughter and hyperactivity. Building on our previous FAST-TRAC studies, the objective of this study is to examine the efficacy of minocycline to improve the cognitive and behavioral deficits of the Angelman Syndrome participant. Currently, the only treatment available for AS patients consists of supportive care including seizure control and behavioral therapy. Our laboratory has collected data indicating improved motor function and cognition after the administration of MC as well as enhanced synaptic function. Further, previously published data suggests the administration of MC to children with other genetically based neurologic disorders (e.g.Fragile X syndrome) dramatically improved dendritic spine morphology and behavioral performance. Taken together, we posit, children treated with MC will experience the same beneficial effects observed in previous studies and the AS mouse model.
Clinical Trial Completed in March 2013

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