Ionis Pharmaceuticals (IONS) said Wednesday it has aligned with the US Food and Drug Administration on ION582, its investigational medicine for the treatment of people living with Angelman syndrome.
"We will enroll a broad group of individuals living with AS in the global pivotal Phase 3 trial, planned to begin in the first half of 2025," said CEO Brett Monia.
Angelman Syndrome is a genetic neurological disease characterized by intellectual disability, impaired verbal abilities, and severe motor impairment.
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