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Evidence Sketchy For BioMarin's Duchenne Drug

This article was originally published in Scrip

Executive Summary

While there may be some evidence suggestive of efficacy with BioMarin Pharmaceuticals Inc.'s experimental drug intended to treat Duchenne muscular dystrophy (DMD) drisapersen, which the company is seeking to market as Kyndrisa, the data are "inconsistent," and in some cases, "contradictory," and do not reach the level of substantial evidence, FDA drug reviewers said in briefing documents released ahead of a Nov. 24 advisory committee meeting.






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