Interesting observation. Is the clinical benefit that has been observed by the trial investigators in the 25mg cohort or the 50mg cohort or if both, which is the optimal biological dose?
One of the other considerations is that the investigator has determined that post trial access is the best medical option for the patient. Presumably, they will have made their request on the basis that termination of the treatment would lead to a deterioration in the patient's overall condition. The results from the original nine patients in the Phase IIa will help inform their decision.
And to the point made by @Mr Perry, avicursen targets CD49d inflammation. The results in December will also help inform the potential of avicursen to treat a wide range of disease indications. There could well be two levels of excitement, the DMD specific and the universal application of avicursen to treat inflammatory disease.
Ann: Percheron launches post-trial access program, page-46
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