exon skipping

/Tag: exon skipping

FDA Approves Drug for Treatment of DMD

Chantal Coles PhD NMDRC provides a Research Update... On September 19th last year the U.S Food and Drug Administration (FDA) approved the first drug for the treatment of Duchenne Muscular Dystrophy, called EXONDYS 51™. This drug, formally known as Eteplirsen, is a gene modifying agent that bypasses a stop codon of the dystrophin, forming a [...]

By | 2018-06-01T03:49:09+00:00 April 3rd, 2017|information, research, trials|0 Comments

Spinal Muscular Atrophy (SMA) Research Update

Spinal Muscular Atrophy (SMA) Research update and treatment - AUSTRALIA A research update by Prof. Monique Ryan Director, Department of Neurology Group Leader, Murdoch Children's Research Institute,  Royal Children's Hospital There has been a lot of activity in the clinical trial space for SMA in the last two years. Two hospitals in Australia- the The [...]

By | 2018-06-01T03:49:11+00:00 November 17th, 2016|atrophies, research, trials|0 Comments

Disappointing Research…

exon skipping trial results released In a press release GlaxoSmithKline and Prosensa have said that the results of the phase 3 exon skipping trial for Duchenne muscular dystrophy are disappointing. Preliminary results show that at the end of the 48 week trial, treated boys were unable to walk any further in six minutes than boys [...]

By | 2017-11-24T11:31:01+00:00 July 23rd, 2016|Comments Off on Disappointing Research…

DMD clinical trial update drisapersen

drisapersen might work after all At the end of 2013 devastating news was announced – the phase 3 clinical trial conducted by GlaxoSmithKline had failed to prove that the exon skipping drug ‘drisapersen’ improved muscle function in boys with Duchenne muscular dystrophy (MD). However, the drug’s original developer – Prosensa – has revealed that, based [...]

By | 2016-09-30T12:33:25+00:00 July 23rd, 2016|Comments Off on DMD clinical trial update drisapersen

Sarepta apply for approval of Duchenne MD drug

Sarepta Therapeutics has issued a press release which gives an update on their plans for the development of exon skipping drug “eteplirsen”. Eteplirsen is a ‘molecular patch’ designed skip a portion of the dystrophin gene called ‘exon 51’. It has the potential to treat around 13 percent of boys with Duchenne muscular dystrophy – those [...]

By | 2018-06-01T03:48:13+00:00 July 23rd, 2016|Comments Off on Sarepta apply for approval of Duchenne MD drug

DMD Duchenne exon skipping trial

Duchenne exon skipping trial results published The results of Sarepta Therapeutics' clinical trial of eteplirsen have been published in a reputable medical journal. Eteplirsen is a ‘molecular patch’ designed skip a portion of the dystrophin gene called ‘exon 51’. It has the potential to treat around 13 percent of boys with Duchenne muscular dystrophy – [...]

By | 2018-06-01T03:48:43+00:00 July 23rd, 2016|Comments Off on DMD Duchenne exon skipping trial