Dutch biopharmaceutical company Prosensa Holding N.V. announced it has been awarded “Best Biotech Pipeline 2013” in the Rare & Orphan Advocacy and Research (ROAR) Awards, which were given for the first time this year.
The inaugural ROAR Awards were announced during the fourth annual World Orphan Drug Congress Europe, which took place Nov. 14-15 in Geneva, Switzerland. The awards have been instituted to recognize and honor the achievements of organizations and individuals from industry, patient and rare disease advocacy communities.
Prosensa’s focus is on rare diseases with high unmet medical need, developing treatments for patients who presently have very limited options. The “Best Biotech Pipeline” award was given in recognition of the broad scope of the company’s orphan drug pipeline, particularly its groundbreaking work in Duchenne muscular dystrophy (DMD), myotonic dystrophy, and Huntington’s disease.
With its primary focus on rare neuromuscular and neurodegenerative disorders with large unmet medical need, Prosensa is engaged in the discovery and development of RNA-modulating therapeutics for the treatment of genetic disorders. The company’s portfolio currently includes six compounds for treating DMD, all of which have been granted orphan drug status in the U.S. and the European Union. Through an innovative technique called exon-skipping, these compounds provide a personalized medicine approach for treating different populations of DMD patients.
For more information, visit www.prosensa.com
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