there is a reason these rare diseases have no treatment so far because it's bloody difficult to tackle! The fact that NNZ2561 has managed to achieve statistically significance across all 4 core measurements is an outstanding result.
Jon said DAYBU only achieved statistically significance on 3 out of 5 core measurements so the result is better than Retts!
Ann: Phase 2 trial shows significant improvements in Pitt Hopkins, page-78
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