This is a phase I/II study. Phase I typically safety, phase II typically safety and dose finding. It was never powered to demonstrate efficacy. It still achieved it's primary end point. Looking for statistical significance on secondary endpoints in a 13 person study is naive.
Unfortunately it also doesn't scream an imminent partnership to fund our completion of Phase III OA.
Ann: Primary Endpoint Achieved in Phase 2 MPS VI Trial, page-41
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