Expert Interview
What Does Sarepta’s Exon 51 Approval Mean for its 9 Other Exon Skipping Product Candidates and Company Future?
Ticker(s): SRPTA muscular dystrophy expert familiar with treating DMD patients and the SRPT trials.
How many MD patients do you currently treat? How many do you treat with DMD?
Added By: kcmckeeWhat are currently your preferred methods for treating patients with DMD? What are their pros/cons?
Added By: kcmckeeWhat are your thoughts on the approval of exon 51? How much of the approval do you think was based on lobbying vs science?
Added By: kcmckeeWhat are your thoughts on the science behind exon 51? Do you see the exon skipping mechanism applicable to genomes other than 51? Why would this be the case?
Added By: kcmckeeHow likely do you think it is that the ongoing clinical trial will demonstrate a clinical benefit to increasing dystrophin production? What could be some potential obstacles SRPT needs to clear?
Added By: kcmckeeWhat are your thoughts on the proposed $300K cost for an annual course of exon 51? Do you see the DMD market growing in line with estimates that see a market of around $1 billion by 2019? What do you see driving this type of growth?
Added By: kcmckeeAre You Interested In These Questions?
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