Expert Interview
Examining Ionis' Phase 1/2a results (six-month data) on ION582 in treating Angelman syndrome
Ticker(s): IONSInstitution: 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: c_adminWhat are your thoughts on the recent data presented on ION582?
Added By: c_adminWhat is your estimate of the current diagnosis rate for Angelman syndrome, and do you think there are many undiagnosed patients, especially among older individuals?
What are the main challenges in diagnosing Angelman syndrome, and how does insurance coverage impact this?
What are the primary manifestations and severity indicators in patients with a maternal deletion in Angelman syndrome?
What do you see as the biggest unmet needs in Angelman syndrome, and how do these vary between communication, seizures, sleep, behavior, and cognition?
How are Angelman syndrome patients currently managed, and does treatment approach vary significantly based on the patient’s age or disease manifestation?
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 and Biogen's ASOs despite promising data?
Given the need for robust clinical trials, what design and endpoints would you recommend for Phase 3 trials of ION582, and do you think a placebo-controlled or sham model is necessary?
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