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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
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.
How many patients do you currently treat with DMD?Added By: joe_mccann
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?
Can you give you high level thoughts on ezutromid? What data is currently published on the drug?
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?
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