The MDEX Consortium was formed to develop and test treatments for Duchenne muscular dystrophy. In the last few years the MDEX Consortium has focussed on a strategy of using patches of genetic material (Antisense Oligonucleotides) to restore production of the essential muscle protein dystrophin. Originally the work of the MDEX Consortium was funded by the Department of Health (UK) in 2005. This project was focused on the search of the most effective molecular patch to promote the restoration of dystrophin production in Duchenne boys carrying specific genetic changes (deletions). This culminated in a phase lb/lla clinical trial in a group of boys who received the molecular patch intramuscularly. This work has been recently completed (http://clinicaltrials.gov/ct2/show/record/NCT00159250). The outcome data of this study has been published in the journal Lancet Neurology (Volume 8, Issue 10, Pages 918 - 928, October 2009). A summary of the outcome can be seen under latest news on this website. A press release relating to this publication and presentation of data, including preliminary data on the current systemic trial at the World Muscle Society (WMS) conference in Geneva, Switzerland in September can be found on http://www.avibio.com/pr/pr439.php.

The activities of the MDEX Consortium are divided into several sections, each led by a different research group (see profiles) but with all members of the Consortium sharing results and meeting regularly. The project also benefits from regular input from a Scientific Advisory Board of experts (see Scientific Advisory Board).

The MDEX Consortium received funding from the UK Medical Research Council (MRC) and AVI BioPharma to perform a clinical trial to assess the safety and efficacy of the same molecular patch utilised in the previous intramuscular study, but this time administered intravenously and repeatedly. Recruitment in London (Great Ormond Street Hospital) and Newcastle (Royal Victoria Infirmary) is now complete.

At the recent TREAT-NMD / NIH International Conference Nov. 17-19 in Brussels, Belgium, data from this clinical study was presented by Professor Francesco Muntoni of the MDEX consortium UK. Data from this ongoing Phase 1b/2 trial at both MDEX sites in the UK demonstrate that AVI-4658 was well tolerated by DMD patients in a dose escalation study that is now up to the sixth and final cohort (20 mg/kg) with no adverse safety signals (see press release on www.avibio.com). The latest data show that systemic treatment with AVI-4658 demonstrates RNA exon skipping and dystrophin protein expression in the first cohorts of DMD patients analysed (see press release on www.avibio.com).

Both study sites are in the UK North Star Clinical network, which links 17 UK centres involved  in the diagnosis and management of DMD (http://www.muscular-dystrophy.org/how_we_help_you/for_professionals/clinical_databases) and would welcome referral of patients for future trials. See the latest news, Q&A in this website and the Clinical Trials website http://clinicaltrials.gov/ct2/show/NCT00844597. Future phase 2b studies are currently in preparation.

The MDEX Consortium along with others, including industry associated in studies on antisense oligonucleotides in DMD met with the representatives of the European Medicines Agency (EMEA) on the 25th September 2009 in London. The purpose of the workshop was to identify pathways that may fast tract this advanced therapy through the regulatory process. A press release by TREAT-NMD relating to this workshop is available on http://www.treat-nmd.eu/patients/news/news/658/

Information on clinical trials in the USA is now available from David Walsey, Head of Invester Relations and Corporate Communications, AVI BioPharma ( This e-mail address is being protected from spam bots, you need JavaScript enabled to view it ).

19.02.2010