By physician referral or invitation only

IONIS Phase 3

To evaluate the efficacy of ION582 in participants with Angelman syndrome (AS) receiving ION582 vs Placebo as measured through expressive communication as well as measured through functional domains, including overall symptoms of disease severity, cognition, communication, sleep, motor functioning, and daily living skills.

Age & Gender

2 years ~ 50 years
Male, Female, Gender Inclusive

Contact the Team

Location

Thank you for your interest, but this study is recruiting by invitation only.

United States (Nationwide)

Additional Study Information

Principal Investigator

Elizabeth Jalazo
Carolina Institute for Developmental Disabilities

Study Type

Clinical or Medical
Interventional

Study Topics

Rare Diseases

IRB Number

25-0577

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