To evaluate the safety and tolerability of ascending dose-levels of multiple intrathecal bolus (ITB) administrations of ION582 in patients with Angelman syndrome (AS) based on incidence and severity of treatmentemergent adverse events (TEAEs) and serious adverse events (SAEs), changes in vital signs and changes in clinical laboratory results.
Thank you for your interest, but this study is recruiting by invitation only.
United States (Nationwide)
Jamie Capal
Carolina Institute for Developmental Disabilities
Clinical or Medical
Interventional
Behavior
Developmental
Rare Diseases
21-2773
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