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exon skipping

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Spinal Muscular Atrophy (SMA) Research Update

By | November 17th, 2016|atrophies, research, trials|

Spinal Muscular Atrophy (SMA) Research update and treatment - AUSTRALIA A research update by Prof. Monique Ryan Director, Department of Neurology Group Leader, [...]

Disappointing Research…

By | July 23rd, 2016|

exon skipping trial results released In a press release GlaxoSmithKline and Prosensa have said that the results of the phase 3 exon skipping [...]

DMD clinical trial update drisapersen

By | July 23rd, 2016|

drisapersen might work after all At the end of 2013 devastating news was announced – the phase 3 clinical trial conducted by GlaxoSmithKline [...]

Sarepta apply for approval of Duchenne MD drug

By | July 23rd, 2016|

Sarepta Therapeutics has issued a press release which gives an update on their plans for the development of exon skipping drug “eteplirsen”. Eteplirsen [...]

DMD Duchenne exon skipping trial

By | July 23rd, 2016|

Duchenne exon skipping trial results published The results of Sarepta Therapeutics' clinical trial of eteplirsen have been published in a reputable medical journal. [...]