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Expert Interview

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

A Second Look: Discussing Denali Therapeutics’ investigational brain-penetrant enzyme therapy, tividenofusp alfa, for mucopolysaccharidosis type II (Hunter syndrome)

Ticker(s): DNLI, TAK

Who's the expert?

Pediatric geneticist, metabolic disease specialist, or pediatric neurologist with experience managing patients with Hunter syndrome / MPS II and familiarity with enzyme replacement therapy and CNS disease progression.

Interview Questions
Q1.

How are you currently managing patients with Hunter syndrome, and what limitations do you see with existing therapies in addressing neurologic versus peripheral disease manifestations?

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Q2.

What is your interpretation of the clinical data for tividenofusp alfa, particularly the reported effects on CNS biomarkers, peripheral biomarkers, and clinical measures such as cognition, adaptive behavior, hearing, or organ involvement? 

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Q3.

How clinically meaningful do you believe a brain-penetrant enzyme replacement therapy could be for patients with MPS II, especially if initiated before advanced neurologic impairment?

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Q4.

How do you expect to discuss the option of switching with your patients? Do you expect to have these discussions with stable patients? Right away? At annual check-in?

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Q5.

Do you have any concerns about antibody development/Neutralizing antibodies that could impact the efficacy of Avlayah? How will that be assessed?

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Q6.

What is on your "checklist" for evaluating patients for whether they should switch to Avlayah

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Q7.

will you proactively call in patients for an appointment to switch them or wait for their annual visit - especially if they don't reach out to you immediately

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Q8.

Do you expect any of your patients remaining on Elaprase? Which patients? Why?

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Q9.

Do geneticists at small community hospitals have familiarity around new therapies? Do they treat Hunter patients or refer them out to COEs?

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Q10.

How concerning are the precautions around membranous nephropathy and anemia listed on the label?

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Q11.

What additional data do you wish/hope to have to support your decision to prescribe Avlayah?

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Q12.

Can a patient return to Elaprase if they can not tolerate Avlayah? Will antibodies make that impossible?

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Q13.

Are IRRs a concern with Avlayah?

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Are You Interested In These Questions?

Slingshot Insights Explained
Call Date
May 07, 2026
Call Time
08:00 AM EDT
Tentative Call Date/Time

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Reason

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