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Prosensa Receives Rare Disease Company Award from EURORDIS

Prosensa Receives Rare Disease Company Award from EURORDIS

Leiden, The Netherlands – 27 February 2013 – Prosensa, the Dutch biopharmaceutical company focusing on RNA-modulating therapeutics for rare diseases with high unmet need, received the ‘Company Award’ at the EURORDIS Black Pearl Gala Dinner in Brussels last night.

The Black Pearl Gala Dinner is hosted annually by EURORDIS, a non-governmental alliance of patients and patient organizations active in the field of rare diseases, to celebrate Rare Disease Day, which takes place on the last day of February. Awards recognize outstanding patients' advocacy groups, volunteers, scientists, companies, media, and policy makers who have contributed to reducing the impact of rare diseases on people's lives.  Proceeds from the event are used to support initiatives to empower leaders of the rare disease community, reduce isolation of people affected by rare diseases, stimulate cutting-edge research and increase public awareness of rare diseases.

Yann Le Cam, EURORDIS CEO, commented: "We are honored to acknowledge the commitment and achievements of this year’s recipients of the EURORDIS Awards. Each of this year’s awardees contributes in their own unique way toward fostering the goals of cooperation embodied in this year’s Rare Disease Day slogan: Rare Disorders without Borders.” He added: “The EURORDIS Company Award recognizes Prosensa’s efforts in developing innovative medicinal products for rare diseases, partnering with major pharmaceutical companies and academic centers, promoting development and access of orphan drugs in Europe, and last - but not least - engaging into a dialogue with patient advocacy groups."

Hans Schikan, Prosensa CEO, commented: “This award from EURORDIS is important to the team at Prosensa. It recognizes the progress we have made to date and reaffirms our commitment to developing personalized medicines for rare diseases for the benefit of patients.”  He added: “We have transformed from a biotech start-up to a rare diseases biopharmaceutical company, thanks to our partnerships with patient organizations, academia and pharma companies.”

Prosensa’s current portfolio includes six compounds for the treatment of Duchenne muscular dystrophy (DMD), all of which have received orphan drug status in Europe and the US. These compounds use an innovative technique called exon-skipping to provide a truly personalized medicine approach to treat different populations of DMD patients. Prosensa’s lead DMD compound, drisapersen, is in Phase III clinical trials in collaboration with GlaxoSmithKline.

Notes to Editors

About DMD
Duchenne muscular dystrophy (DMD) is a severely debilitating childhood neuromuscular disease that affects 1 in 3,500 live male births. This rare disease is caused by mutations in the dystrophin gene, resulting in the absence or defect of the dystrophin protein. As a result, patients suffer from progressive loss of muscle strength, often rendering them wheelchair-bound before the age of 12. Respiratory and cardiac muscle can also be affected by the disease and most patients die in early adulthood due to respiratory and cardiac failure.

About exon skipping
The dystrophin gene is the largest gene in the body, consisting of 79 exons. Exons are small sequences of genetic code which, via an intermediate step involving RNA, lead to the assembly of sections of protein. In DMD, when certain exons are mutated/deleted, the RNA cannot read past the fault. This prevents the remainder of the exons from being read, resulting in a non-functional dystrophin protein and the severe symptoms of DMD. RNA-based therapeutics, specifically antisense oligonucleotides inducing exon skipping, are currently in development for DMD. These antisense oligonucleotides skip an exon next to a defective exon and thereby correct the reading frame, enabling the production of a novel, functional dystrophin protein. Prosensa’s exon skipping technology was licensed from Leiden University Medical Center.

About Prosensa
Prosensa is an innovative Dutch biopharmaceutical company focused on the discovery, development and commercialization of RNA-modulating therapeutics correcting gene expression in diseases with significant unmet need, in particular neuromuscular disorders. Prosensa’s current focus is on developing treatments for Duchenne muscular dystrophy (DMD), myotonic dystrophy and Huntington’s disease. In 2009, Prosensa entered into a strategic alliance for part of its DMD exon skipping program with GlaxoSmithKline. Prosensa’s lead compound, drisapersen (GSK2402968/ PRO051), being developed by GSK, is in Phase III clinical trials. Prosensa is a privately held biopharmaceutical company, backed by a consortium of Abingworth, Gimv, Idinvest Partners, Life Sciences Partners, MedSciences Capital and New Enterprise Associates. For more information, please visit

Issued for and on behalf of Prosensa.

To contact the Prosensa team at College Hill, email

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Melanie Toyne-SewellManaging Partner