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Prosensa was a Dutch biopharmaceutical company dedicated to the discovery and development of RNA-modulating therapeutics for rare genetic diseases, with a primary focus on Duchenne Muscular Dystrophy (DMD). The company utilized a proprietary exon-skipping platform to develop antisense oligonucleotides designed to restore the production of functional dystrophin protein. Its lead product candidate, drisapersen, was one of the first potential disease-modifying treatments for DMD to reach late-stage clinical trials. Prosensa established a significant partnership with GlaxoSmithKline before regaining full rights to its portfolio and eventually being acquired. In 2015, the company was acquired by BioMarin Pharmaceutical for approximately $680 million upfront to strengthen its rare disease pipeline.
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