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Leiden, The Netherlands, Jan. 13, 2014 (GLOBE NEWSWIRE) -- Prosensa Holding N.V. (NASDAQ: RNA), the Dutch biopharmaceutical company focusing on RNA-modulating therapeutics for rare diseases with high unmet medical need, will be providing updates to both the financial and patient communities regarding drisapersen.
As previously announced, the company will be providing a full corporate update, which will now also include additional analyses of the drisapersen dataset, at the 32
nd Annual J.P. Morgan Healthcare Conference on Thursday, January 16, 2014 at 10:00 AM Pacific Time. Furthermore, the company will be providing a separate update to the patient community on Tuesday, January 21, 2014 at 8:00 AM Eastern Time. A live webcast of these presentations can be accessed through the Investors & Media section of the Prosensa corporate website at
http://ir.prosensa.eu/events.cfm and will be archived for 90 days.
About drisapersen and the clinical development program
Drisapersen, (previously GSK2402968/PRO051), an antisense oligonucleotide which induces exon skipping of exon 51, is currently in-late stage development for DMD. Drisapersen has orphan drug status in the EU, US, Japan and Australia. In June 2013, drisapersen was granted Breakthrough Therapy designation by the US Food and Drug Administration.
The overall drisapersen clinical registration program comprises three double-blind, placebo-controlled studies (DMD114117, DMD114876 and DMD114044) and two long term open-label extension studies (DMD114673 and DMD114349).
Duchenne Muscular Dystrophy (DMD) is a severely debilitating childhood neuromuscular disease that affects up to 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. Patients suffer from progressive loss of muscle function, often making them wheelchair bound before the age of 12. Respiratory and cardiac muscle can also be affected by the disease. Few patients survive the age of 30.