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Despite 21st Century Cures Act victory, the road forward is murky for rare disease community

By Marie Powers
News Editor

Monday, February 13, 2017

As Rare Disease Day approaches, there's plenty to celebrate. The acquisition of Baxalta Inc. by Shire plc early in 2016 created a global giant with an equally large appetite for therapies to treat rare indications. Last year saw a huge, albeit controversial, breakthrough to treat Duchenne muscular dystrophy (DMD) with accelerated approval of Sarepta Therapeutics Inc.'s exon-skipping drug, eteplirsen, branded Exondys 51. And as the year ended, passage of the 21st Century Cures Act – encompassing patient-focused drug development, qualification of biomarkers and other development tools, and novel clinical trial designs – became a legislative triumph for the rare disease community.

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