Portrait Prosensa is a Dutch biopharmaceutical company dedicated to the development of RNA-based therapeutics targeting diseases with unmet medical needs, in particular neuromuscular disorders. Prosensa's drug development is based upon a unique and proprietary technology platform involving 'exon skipping', enabling correction of mutated RNA. This ability to modulate genes selectively through RNA-based therapeutics could provide a new way to treat a wide range of human diseases. The company has a leadership position in fundamental patents, technology, and know-how relating to RNA-based approaches and exon skipping. Prosensa's lead compounds for DMD are currently in clinical phase I/II development and the company has received orphan drug designation both in Europe and the US.
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