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Expert Interview

Slingshot members are talking to an expert! The topic is:

Transformative Therapeutic Breakthrough: Unveiling the Potential of Ultragenyx Pharmaceutical Inc.'s GTX-102 in Angelman Syndrome Treatment

Ticker(s): RARE

Who's the expert?

A neurologist or pediatrician specializing in neurodevelopmental disorders, particularly Angelman syndrome. The expert should possess in-depth knowledge of the current therapeutic landscape for Angelman syndrome, as well as an understanding of the biological mechanisms targeted by GTX-102.

Interview Questions
Q1.

Considering the interim data, could you elaborate on the multidomain improvements observed in patients treated with GTX-102, particularly in cognition, behavior, and motor function?

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Q2.

How do the improvements observed in the GTX-102 study compare with the natural history data of Angelman syndrome, especially in terms of cognitive and motor milestones?

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Q3.

The ABC-C assessments showed rapid improvement in hyperactivity and noncompliance. How significant are these findings in improving the quality of life for patients and their caregivers?

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Q4.

The long-term data from Dose-escalation Cohorts demonstrate sustained clinical benefits exceeding Natural History data at Day 758. What implications does this have for the durability of GTX-102 treatment effects?

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Q5.

Dr. Sell mentioned functional gains such as improved communication and motor skills in patients. How do these gains impact the daily lives and independence of individuals with Angelman syndrome?

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Q6.

The press release mentions mild to moderate adverse events related to lower extremity weakness. Could you provide insight into the safety profile of GTX-102 and its implications for ongoing treatment?

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Q7.

What are the key considerations for transitioning GTX-102 into a Phase 3 pivotal study, and how might regulatory interactions influence the future development of this therapy?

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Q8.

With the diverse manifestations of Angelman syndrome, how do you envision GTX-102 being tailored to address individual patient needs and genetic variations in future clinical practice?

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