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Expert Interview

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

After a roller coaster year for DMD treatments and the recent granting of Rare Pediatric Disease designation from the FDA for ezutromid does it have potential in DMD?

Ticker(s): SMMT, SRPT, BMRN, PTCT

Who's the expert?

A DMD expert that has been closely involved or aware of the recent developments and news at the agency. The expert should treat a number of patients and be aware of the therapy ezutromid.

Interview Questions
Q1.

How many patients do you currently treat with DMD?

Added By: joe_mccann
Q2.

Please discuss the primary endpoint:
Change from baseline in MRI parameters related to fat infiltration and inflammation in leg muscles
Is this endpoint a surrogate endpoint in your mind? How does it relate to the FDA panel on 4/25 questioning 6mw as a valid endpoint?



Added By: joe_mccann
Q3.

Can you give you high level thoughts on ezutromid? What data is currently published on the drug?

Added By: joe_mccann
Q4.

It looks like ezutromid works by modulating utrophin. How well understood is the role of utrophin in DMD patients? Do we know the role it plays relative to dystrophin?

Added By: joe_mccann

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Reason

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