American biopharmaceutical company at Warren in New Jersey, founded in 1998 and listed on Nasdaq, devoted to rare disease. Develops and markets medicines including ataluren for nonsense mutation Duchenne muscular dystrophy, risdiplam for spinal muscular atrophy discovered with Roche and the SMA Foundation, translarna, emflaza and gene therapy and metabolic disease products from its Bio-e and splicing platforms, and runs its own discovery research, clinical development and commercial operations worldwide.
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