Monday, 24 November 2014

» BioMarin to purchase Prosensa and prioritise licensing for drisapersen Action Duchenne

» BioMarin to purchase Prosensa and prioritise licensing for drisapersen Action Duchenne: "BioMarin Pharmaceutical have agreed to purchase Prosensa for $680 million , with two further payments of $80 million subject to drisapersen , Prosensa’s leading candidate for skipping exon 51 in Duchenne, being licensed in the USA by 15th May 2016 and Europe no later than 15th February 2017.

 

Jean-Jacques Bienaim√©, Chief Executive Officer of BioMarin said “BioMarin is dedicated to the rare disease community, and the acquisition of Prosensa fits strategically with our mission of delivering therapies that address serious unmet medical needs. We are committed to working closely with regulatory authorities worldwide in bringing a potentially breakthrough therapy to patients with this devastating condition.”"



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Thursday, 20 November 2014

Cardiac stem cell treatment restores heart function damaged by Duchenne muscular dystrophy

Cardiac stem cell treatment restores heart function damaged by Duchenne muscular dystrophy: "Researchers at the Cedars-Sinai Heart Institute have found that injections of cardiac stem cells might help reverse heart damage caused by Duchenne muscular dystrophy, potentially resulting in a longer life expectancy for patients with the chronic muscle-wasting disease."



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Thursday, 13 November 2014

» Sarepta Therapeutics Announces First Patient Dosed in Study of Non-Ambulant Patients with DMD Action Duchenne

» Sarepta Therapeutics Announces First Patient Dosed in Study of Non-Ambulant Patients with DMD Action Duchenne: "On the 12th of November, Sarepta announced the initiation for dosing in the 204 clinical study for non-ambulant and advanced DMD patients. The link to the official press release can be found here: http://investorrelations.sarepta.com/phoenix.zhtml?c=64231&p=irol-newsArticle&ID=1989115

This study includes around 20 patients treated with eteplirsen whom can particularly be treated with exon 51 skipping. The investigation is being carried out in the United States and the major aims are to evaluate the safety of eteplirsen in DMD patients over a dosing time period of 96 weeks."



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