Conference 2007 Report
You are now able to download presentations from the conference by logging into our document library.
The conference saw the launch of our new Charity name Action Duchenne with the development of this website. Professor Muntoni from the Hammersmith Hospital kindly opened a bottle of champagne at our dinner to mark the start of the new MDEX trials and the launch of Action Duchenne.
Over 270 people attended the 5th Duchenne International conference in London this year. The conference called “A change is gonna come” marked yet further strides being made in research for new treatments and clinical trials.
Prosensa in Holland and the MDEX team with AVI are moving ahead with clinical trials using antisense oligonucleotides to skip faults in the gene. Although these are early safety trials they represent the possibility of a therapy for Duchenne. Steve Wilton again delighted the conference with his detailed explanation of exon skipping and how all boys could potentially be treated bar a small minority. Matthew Wood developed a very interesting proposal on using PNA’s as a potential molecule for exon skipping in Duchenne.
PTC Therapuetics also reported on further trials for their drug PTC124 that is hoped will skip through premature stop codons present in about 10% of those living with Duchenne.
Summit’s leading scientist Jon Tinsley explained that the new drug SUMC1100 a Utrophin upregulator will start clinical trials next year. Summit also announced a new project with Action Duchenne the ZF Partnership and we were able to present to Jon a cheque for £279,000 with major donors The Gavriel Meir Trust, Action Duchenne and Charleys Fund. Tracy Levene from Manchester announced a further contribution of £50,000 towards this new drug discovery programme. The screens using zebrafish embryos and other screens have already come up with new potential drugs for Duchenne. So this is really exciting cutting edge research funded and promoted by Action Duchenne.
There was great interest in Ronald Cohn’s mouse trial on Losartan and he is about to start a human trials to see if this might help boys with Duchenne. He advised everyone, however, to wait until he had these results before using Losartan.
Stem cell research is progressing and although Yvan Torrente was unable to attend we had updates from Jenny Morgan and Terry Partridge.
There were excellent presentations on ventilation, steroids and physiotherapy. John Bourke announced the imminent start of his 5 year trial on heart drugs that might help to reduce the effects of cardiomyopathy.
John Joe Bardsley the director of the Channel 5 Extraordinary lives programme The boy who will never grow up and Stuart who is living with Duchenne attended the conference and spoke about the film and its message. The delegates stood to applaud the film, Stuarts wonderful contribution and to celebrate the life of Stuarts friend Harry who sadly died only two days before.
Robin Sharp reported on changes being made to the DMD Registry and Steve Abbs spoke about the need for high quality genetic testing. There will be a new online registration process and the Registry will be bought in line with the new Treat NMD Charter for patient registries.
Miranda Grounds from Australia reported on new approaches to using drugs and supplements for Duchenne and Nic Wells summarised new strands of research in Gene Therapy. George Dickeson explained how the use of AAV viral vectors could have potential therapeutic benefit for Duchenne.
Kate Bushby spoke about the excellent new European wide Treat NMD initiative and Mike Hanna about the new MRC translational research centre at Great Ormond Street /UCl/Newcastle.
Why not go to our document library to see all of their presentations?
Also we will have written reports on the whole conference and the Exon skipping workshop and young people’s forum published in the next few months on the website.
Conference 2007
