New Ataluren UK Clinical Trials - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

New Ataluren UK Clinical Trials - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: We are delighted to inform you that PTC Therapeutics has been able to establish an extension study in the UK for their drug ataluren for patients who participated in the Phase 2b. This extension study will take place at two centres: Newcastle and London. The background to this decision by PTC Therapeutics can be found at http://www.actionduchenne.org/viewarticle?news=387 .
Secondly, PTC Therapeutics is in active communication with European regulators to run a wider confirmatory study with the aim of confirming the findings of the previous study. We look forward to welcoming PTC Therapeutics to our forthcoming annual conference where we hope there will be an opportunity to find out more about their plans.

First approval for AAV vector gene therapy - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

First approval for AAV vector gene therapy - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: Netherlands biotech company UniQure have announced that Glybera has become the first gene therapy approved by regulatory authorities in the Western world.

It is the first medication approved for patients with rare metabolic disorder Lipoprotein Lipase Deficiency and commercial roll-out to begin second half of 2013. The company that has taken this product over from AMT state that it now validates uniQure’s unique AAV-based gene therapy platform.

Prosensa Announces Progress on Exon Skipping Compounds for the Treatment of Duchenne Muscular Dystrophy - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

Prosensa Announces Progress on Exon Skipping Compounds for the Treatment of Duchenne Muscular Dystrophy - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: Prosensa has also received orphan drug designation by the European Commission for PRO045 and PRO053. These compounds are designed to skip exons 45 and 53 of the dystrophin gene and are expected to enter clinical development within the next 6 months.

In A First, An Experimental Drug May Help Boys With Muscular Dystrophy - Forbes

In A First, An Experimental Drug May Help Boys With Muscular Dystrophy - Forbes: A new experimental drug appears to improve the muscle function of boys with muscular dystrophy, increasing the distance the boys can walk in six minutes compared to when they began taking it a year before.

DMD: Reformulated Utrophin Upregulator Looks Good in Healthy Volunteers | Quest Magazine Online

DMD: Reformulated Utrophin Upregulator Looks Good in Healthy Volunteers | Quest Magazine Online: Summit PLC, a drug discovery company based in Oxford, U.K., has announced that SMT C1100, its experimental oral drug for Duchenne muscular dystrophy (DMD), appears safe and well-tolerated in healthy volunteers at all doses tested, and that it reaches levels in the bloodstream believed to be adequate for a therapeutic benefit.

Positive Phase 1 trial results for SMT C1100 (Summit PLC) - PPMD Community

Positive Phase 1 trial results for SMT C1100 (Summit PLC) - PPMD Community: Following closely on the heels of the good news about Sarepta’s exon 51 skipping strategy, PPMD is pleased to report that Summit PLC�has been able to achieve sufficient plasma levels of its reformulated utrophin-upregulating drug to justify a phase I/II trial in Duchenne (Read Summit's Press Release). At PPMD we have always believed in “hedging our bets” and investing widely in different therapies, some of which are mutation specific and some of which are not. Unlike exon-skipping strategies, upregulating utrophin is not a mutation specific strategy and, if effective, could potentially benefit anyone with Duchenne

Promising News from Sarepta on Exon 51 Skipping Trial - PPMD Community

Promising News from Sarepta on Exon 51 Skipping Trial - PPMD Community: The much-anticipated 48-week results on eteplirsen, a drug designed to skip exon 51, were presented today by Sarepta Therapeutics�on a webinar and press release. On the webinar, Sarepta’s Chief Executive Chris Garabedian presented data about the dystrophin found in study participants’ muscle biopsies and the 6-minute walk results.