- Trials with a EudraCT protocol (1)
- Paediatric studies in scope of Art45 of the Paediatric Regulation (0)
1 result(s) found for: Ubiquitin ligase.
Displaying page 1 of 1.
EudraCT Number: 2021-003009-23 | Sponsor Protocol Number: ION582-CS1 | Start Date*: 2022-07-19 | |||||||||||
Sponsor Name:Ionis Pharmaceuticals, Inc. | |||||||||||||
Full Title: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION582 in Patients with Angelman Syndrome | |||||||||||||
Medical condition: Angelman syndrome is a neurogenetic disorder caused by a loss of function of the maternally inherited UBE3A gene (chromosome 15) which codes for UBE3A ubiquitin protein ligase. AS presents early in... | |||||||||||||
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Population Age: Children, Adolescents, Under 18, Adults | Gender: Male, Female | ||||||||||||
Trial protocol: FR (Trial now transitioned) IT (Trial now transitioned) NL (Ongoing) | |||||||||||||
Trial results: (No results available) |
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