Leiden, The Netherlands – 15 November 2013 – Prosensa Holding N.V. (NASDAQ: RNA) the Dutch biopharmaceutical company focusing on rare diseases with a high unmet medical need, has been awarded Best SME Biotech Pipeline 2013 in the Rare & Orphan Advocacy and Research (ROAR) Awards.
The ROAR Awards were announced during the 4th Annual World Orphan Drug Congress Europe, November 14-15, 2013, in Geneva, Switzerland. The inaugural 2013 awards are an opportunity to recognize and honor achievements by organizations and individuals from industry, patient, and rare disease advocacy communities.
"There is a desperate unmet need in rare diseases, and we are dedicated to developing treatments for patients who face very limited options," said Hans Schikan, CEO of Prosensa. "We are honored to accept this award and would like to thank the judging committee for recognizing our efforts, and our many partners and advocates for their steadfast support."
The Best SME Biotech Pipeline Award recognizes the breadth and depth of the company's orphan drug pipeline, most notably its pioneering work in Duchenne muscular dystrophy, myotonic dystrophy and Huntington's disease.
Notes to editors
About Prosensa Holding N.V.
Prosensa (NASDAQ: RNA) is a Dutch biotechnology company engaged in the discovery and development of RNA-modulating therapeutics for the treatment of genetic disorders. Its primary focus is on rare neuromuscular and neurodegenerative disorders with a large unmet medical need, including Duchenne muscular dystrophy, myotonic dystrophy and Huntington's disease.
Prosensa's current portfolio includes six compounds for the treatment of DMD, all of which have received orphan drug status in the United States and the European Union. The compounds use an innovative technique called exon-skipping to provide a personalized medicine approach to treat different populations of DMD patients.
Issued for and on behalf of Prosensa.
To contact the Prosensa team at College Hill, email Prosensa@collegehill.com