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A biopharmaceutical company developing therapeutic products principally based on third-generation NEUGENE antisense technology.
Congratulations to Sarepta (formerly AVI) for bringing a very promising technology one step closer to becoming a cure. My red thumb in CAPS marks what I believe is an exaggerated emotional reaction to a great result in the phase IIb trial (I believe the causes of this reaction has 2 components: Wall Street's interest in making billion-dollar companies, and the emotional need of many people, including myself, to help these extremely unfortunate boys). The importance of the fact that eteplirsen has shown no side effects thus far cannot be overstated, of course, but I still see a long path prior to regulatory approval. This is one of the few cases where I wish I am wrong in CAPS.
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