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SKIP-NMD clinical trial now progressing to part 2

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 The SKIP-NMD team successfully completed the first part of the clinical trial, a dose escalation phase with no significant problem for the 12 children recruited so far, and recently obtained the go-ahead from the data safety monitoring board to initiate the second, maintenance  phase of the study.

Sarepta Therapeutics Announces First Patient Dosed in European Phase I/II Study of SRP-4053 in Duchenne Muscular Dystrophy Patients

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 The full press release, including a printer-friendly version can be found on the company website:

Information regarding the SKIP-NMD clinical trial of Sarepta’s drug candidate SRP-4053 is now available on

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Eligibility criteria and other information regarding the clinical trial of Sarepta’s drug candidate SRP-4053, for eligible patients with Duchenne muscular dystrophy is now available on by searching “NCT02310906” in the search box.

SKIP-NMD Webinar now available

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The SKIP-NMD webinar took place on 8th September at 17.00 BST in the UK and in Italy at 17.30 CEST.
The webinar was organised by the patient and family representatives in SKIP-NMD to inform the patient community about exon-skipping trials in general and SKIP-NMD in particular.

The videos in English and Italian are available here:

SKIP-NMD Webinar for DMD families on 8th September

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A webinar has been organised by the patient and family representatives in SKIP-NMD to inform the patient community about exon-skipping trials in general and SKIP-NMD in particular. This will take place in the UK on 8th September at 17.00 BST and in Italy at 17.30 CEST.

SKIP-NMD Clinical Trial Applications submitted

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The Clinical Trial Applications for SKIP-NMD clinical trial (4053-101) have been submitted for review by Sarepta Therapeutics in applicable countries. It is expected that enrollment will begin in September, with dosing to begin in October or November.

World Duchenne Awareness Day

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 The first World Duchenne Awareness Day will take place on 7 September 2014. Duchenne patients and organisations all over the world will participate in order to raise more awareness for Duchenne Muscular Dystrophy.

Members of the Skip-NMD consortium met in Rome on 2nd-3rd June 2014 for the scheduled 6 monthly meeting.

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The meeting, which coincides with the intermediate progress of the grant, was attended by more than 30 members of the consortium which include clinicians, scientists, radiologists and physicists, coordinators, therapists, ethicists, representatives from support groups from the 3 countries involved (UK; France and Italy) and from the industrial partner Sarepta Therapeutics from USA.

18th International World Muscle Society Congress, 1-5th October 2013, California USA

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The 18th International World Muscle Society Congress will be held on 1-5th October 2013 in California USA.

SKIP-NMD advertised in the on-line TREAT-NMD newsletter

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TREAT-NMD is a network for the neuromuscular field that provides an infrastructure to ensure that the most promising new therapies reach patients as quickly as possible. Since its launch in January 2007 the network's focus has been on the development of tools that industry, clinicians and scientists need to bring novel therapeutic approaches through preclinical development and into the clinic, and on establishing best-practice care for neuromuscular patients worldwide.

SKIP-NMD Bi-annual Meeting

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SKIP-NMD partners meet up twice a year to discuss the project progress.

The second project meeting will be held in 11-12 July 2013 at the Institut de Myologie in Paris, France.

Drisapersen (skips exon 51) US phase II clinical trial

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GSK announced in Jan 2013 that they had recruited their last patient for the drisapersen (skips exon 51) US phase II clinical trial which will be completed in November 2013 and the results available in early 2014.

GSK announces support for AllTrials campaign for clinical data transparency

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The Alltrials campaign is calling for registration of clinical trials and the disclosure of clinical trial results and reports in the public domain. GSK has now announced that it supports this.

European Neuromuscular Centre (ENMC) workshop

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 European Neuromuscular Centre (ENMC) workshop on clinical applications of anti-sense mediated exon-skipping for DMD was held in December 2012 in Naarden, The Netherlands.

Rare Disease Day 2013

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The SKIP-NMD grant was selected by the European Commission to feature in its news release on Rare Disease Day 2013 (Feb 18th) as one of four FP7 projects it recently funded in rare disease research.

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