The main goal of the study is to evaluate the safety and efficacy of GTX-102 in participants with Angelman syndrome.
Signed informed consent from parent(s) or legal guardian(s)
Subprotocol A: ≥ 1 to < 4 years of age with a genetically confirmed diagnosis of deletion-type Angelman syndrome
Subprotocol B: ≥ 4 to < 18 years of age with a genetically confirmed diagnosis of UPD/ICD Angelman syndrome
Subprotocol C: ≥ 18 to < 65 years of age with a genetically confirmed diagnosis of Angelman syndrome, any genotype
Any change in medications or diet/supplements intended to treat symptoms of Angelman Syndrome (eg, sleeping aids, antiseizure medications, supplements, dietary change including ketogenic or low-glycemic index diet, other) within the month prior to the Screening Visit (excluding weight-based adjustments)
Any condition that creates an increased risk of unsuccessful lumbar puncture
Current or expected concomitant use of drugs that increase the risk of bleeding (eg, heparin, low molecular weight heparin, platelet inhibitors)
Known hypersensitivity to GTX-102 or its excipients or required premedication that, in the judgment of the Investigator, places the subject at increased risk for adverse effects