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Orphan Drug Trailblazers

trailblazing_in_a_wheelchaiThis week I’d like to shine the spotlight on a few companies who are making a difference in the field of orphan drug development.

1. PSR-Agility, a leading Dutch orphan-drug development expert, has been nominated for the category “Best CRO for Orphan Drugs” in the ROAR Awards.

2. On November 7 Prosensa, the Dutch biopharmaceutical company which focuses on RNA-modulating therapeutics for the treatment of rare diseases, announced the successful enrollment of the 100th patient into their Natural History study of Duchenne muscular dystrophy (DMD).

3. The FDA announced on November 1 that Roche’s Gazyva (obinutuzumab) has been approved for the treatment of patients with previously untreated chronic lymphocytic leukemia (CLL), and that Gazyva has been

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granted orphan drug designation.

Kudos to these companies who think outside the box, blaze new trails and change the blockbuster-dominated landscape to give hope to patients who don’t get a lot of good news.