In Research
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April 2008 MDEX News
Clinical Trials:
AVI 4658, a morpholino antisense oligomer to induce exon 51 skipping.
The first two patients tolerated well the intramuscular injection of the morpholino antisense. Data from these 2 patients were discussed with the Safety Monitoring Committee (SMC) on 11th March 2008 who were pleased with the reports and the lack of any adverse events relating to AVI 4658. However, in line with all studies involving drug manufacturers, outcome data on the current clinical trial is confidential and will not be released until the trial is completed.
Following discussion with the Safety Monitoring Committee and members of the MDEX consortium, it was decided to file some essential protocol amendments ahead of the recruitment of the next patients. These amendments to the protocol are being submitted to the regulatory body MHRA.
AVI BioPharma is in the final phase of submitting an application to MHRA for the systemic exon 51 PMO Trial which will be performed in collaboration with the MDEX consortium.
PTC 124, a small molecule drug taken orally has the potential to treat genetic disorders that carry a nonsense mutation. Approximately 10% of DMD boys carry nonsense mutations. A Phase 2b trial in Europe on DMD and BMD subjects is due to commence later this year. Newcastle, Oswestry and London ICH/GOSH will participate in this trial. All three study centres have site approval from NREC for the trial. PTC Therapeutics held an Investigator meeting in Newcastle on 28-29 February 2008. All three
MDI
Discussions are ongoing between the MDEX consortium and MDI for further funding, preferably involving clinical trials.
TREAT-NMD AON workshop
A workshop to update studies on AON design and systemic delivery will be held between invited scientist of TREAT-NMD and the MDEX consortium on 10th May 2008 at ICH in London.
The Move
The move of the Neuromuscular Unit (NMU) from Imperial College London to the UCL Institute of Child Health (ICH) and GOSH commenced on the 1st December 2007 with Francesco’s appointment with ICH.
All muscle pathology, laboratory researcher and clinical personnel have now moved to the new site.
Francesco’s new contact details are as follows:
Address: Dubowitz Neuromuscular Centre, 1st Floor Institute of Child Health, 30 Guildford Street, London WC1N 1EH.
Tel: 020 7905 2111 Fax: 020 7905 2832 email: f.muntoni@ich.ucl.ac.uk
The standard email format for staff who moved to the ICH is <initial.surname@ich.ucl.ac.uk>
Your comments would be welcome and any news that you would like to include in the May issue should be sent, latest a few days before the end of the month.
Please note my change of email address to k.ganeshaguru@ich.ucl.ac.uk