Given that the first patient was dosed over 4 months ago, the investigators would have obtained a lot of data to date including key data on efficacy. Upper limb assessment is scheduled during treatment weeks 5, 8, 12 and 24. One would assume that week 12 would be particularly informative in terms of interim results in the open label trial.
Are the week 12 results to date on upper limb performance encouraging or disappointing? What is encouraging is that PPMD has chosen to incorporate details of the ATL1102 for DMD on their website at this relatively late junction in the trial. Perhaps its to get the last enrolments across the line or perhaps its because.....well its all so encouraging.
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- Ann: Phase II clinical trial in DMD Update
Ann: Phase II clinical trial in DMD Update, page-20
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