I visited ACTION DUCHENNE site for conference details.

For various scientific sessions which are planned in NOV, they had prepared small document giving details of each presentation/session/speaker.


I could gather these information which in certain cases I was not aware of.


Related to Professor Steve Wilton work

The State Government of Western Australia has provided funds to establish a on-stop muscular  dystrophy clinic for DMD in anticipation

of commencing clinical trials in 2012.

Initial trials will involve non-ambulant patient, to address safety issues, and then we hope to first address any patient whose mutation

may be responsive to splice switching intervention. Local boys will be seen first but we hope to transfer materials to other clinics across Australia and the world as resources permit.


Our goal is to test biglycan therapy in clinical trials. We are aiming to initiate testing in three years or less.



Completion of the study will depend on when the necessary funding is secured. Once the funding is in place, the Phase I trial in healthy volunteers could be completed within twelve months.


Related to Dr Mike Gait and Professor Matthew Wood work

We hope to continue for some time to develop peptides suitable for use with different diseases and different antisense oligonucleotide

types, but we believe that a candidate peptide for clinical development in DMD is within months of being finalised.


Related to Professor George Dickson Royal Holloway College London work

We are not yet at the clinical trial stage but the therapy should be appropriate for all DMD patients. Initial safety trials would probably

initially recruit older boys with DMD for ethical reasons. Ultimately treatment would be delivered at as young an age as safely acceptable.




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