HIGHLIGHTS
- who: L. R. Ranganath from the (UNIVERSITY) have published the Article: Clinical development innovation in rare diseases: overcoming barriers to successful delivery of a randomised clinical trial in alkaptonuriau2014a mini-review, in the Journal: (JOURNAL)
SUMMARY
At the same time, these aspects also require a large sample size for statistical power of the study despite the rarity of AKU, a significant challenge. Initial studies employed an oral dose of 2 mg daily even though a full dose-response profile was never undertaken, and adopted for the first NIT interventional study; 20 NIT-treated . . .
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