Expert Interview
Angelman Syndrome Pipeline: Recent Updates on GTX-102 (Ultragenyx & GeneTx), ION582 (Ionis Pharmaceuticals), and rugonersen (Roche).
Ticker(s): RARE, IONS, RHHBYInstitution: University of California San Diego
- Dysmorphologist and clinical geneticist at Rady Children's, and a professor of clinical pediatrics at UC San Diego.
- Medical director of Rady Children's multidisciplinary Prader-Willi Syndrome Clinic and co-founder and medical administrator of Rady Children's Down Syndrome Center.
- Research focuses on Angelman syndrome, and she is the principal investigator in San Diego of a multi-site consortium that conducts a natural history study and clinical trials.
Roughly how many patients with Angelman syndrome do you currently manage?
Added By: sara_adminWhat are your thoughts on the recent data presented on ION582?
Added By: sara_adminWhat are your thoughts on the recent data presented on GTX-102?
Added By: sara_adminWhat are the main challenges in diagnosing Angelman syndrome, and how does insurance coverage impact this?
What are your thoughts on the safety and efficacy of ION582 compared to other ASOs for Angelman syndrome, and what might explain the discontinuation of Roche's rugonersen despite promising data?
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