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November 2007 MDEX Newsletter
Welcome to the November MDEX Newsletter. This monthly Newsletter is intended to update all within the MDEX consortium about news and activities in the past month.
Clinical Trial
The trial is now recruiting. The first patient who was consented on Friday 29th October 2007 is planned to have the antisense oligomer injected before the end of December. Another patient has been contacted and will attend in early December to discuss possible participation to the trial.
MHRA
MHRA held an Investigator’s interview and an inspection of the laboratory facilities involved in the DMD PMO clinical trial CRO490 at Imperial College London Hammersmith Hospital Campus on 28th and 29th November 2007. The interview and the laboratory inspection centered on various issues relating to the conduct of the trial. The trial team felt that all went well and a report from MHRA will be available in due course.
Representatives of AVI BioPharma (Peter O’Hanley & Janet Christensen) and the MDEX Consortium (Francesco Muntoni, Dominic Wells & Steve Wilton) met with MHRA advisers on 6th November 2007. The meeting was to obtain advice on a proposed application to MHRA by AVI BioPharma for a systemic phase I/IIa clinical trial in DMD patients with the AVI-4658 PMO.
Press Release and BBC Broadcast
The MDEX agreed version of the press release is now available on the Imperial College London and on the Imperial Healthcare NHS Trust websites.
Enquiries by patients and families of affected individuals are still being received following the BBC broadcast on 19th October 2007, to participate in the exon 51 proof of principle phase l/lla clinical trial.
Publication
Congratulations to the Oxford Group on the following DoH funded publication.
Yin H, Lu Q, Wood M; Effective exon skipping and restoration of dystrophin expression by peptide nucleic acid antisense oligonucleotides in mdx mice; Molecular Therapy, advance online publication, October 2007.
PPUK
The 5th International Duchenne Conference (2-4 November 2007) in London UK, organised by PPUK held a round table workshop on the first day for scientists and clinicians to review current trends in research and treatment of DMD. The workshop was successful and Nick indicated that it will be regular feature in future conferences.
MDI
Following their two successful meetings in October (3rd and 18th) MDI will again meet with representatives of their (Irish) government this week.
SAB – MDEX
A joint meeting of the Scientific Advisory Board (SAB) and MDEX Consortium was held on 16th November 2007 at the Muscular Dystrophy Campaign (MDC) headquarters. An update on the science and the clinical trial was presented and discussed.
The Move
The move of the Neuromuscular Unit (NMU) from Imperial College London to the Institute of Child Health (ICH), UCL commences on the 1st December 2007. Francesco’s appointment with ICH is from 1st December with honorary appointments at Imperial College London and Imperial Healthcare NHS Trust until the move is completed in spring of 2008. The NMU laboratories and associated staff will start their move early in 2008 and will be updated on the MDEX monthly newsletter.
The current phase l/ll clinical trial will continue to be conducted under the auspices of Imperial College London.
Francesco’s new contact details are as follows:
Address: Dubowitz Neuromuscular Unit, 1st Floor Institute of Child Health, 30 Guildford Street, London WC1N 1EH.
Tel: 020 7905 2608 Fax: 020 7905 2832 email: f.muntoni@ic.ac.uk
Your comments would be welcome and any news that you would like to include in the December issue should be sent, latest a few days before Christmas. k.ganeshaguru@imperial.ac.uk