A Father’s Fight to Help His Sons–And Fix Clinical Trials

January 9, 2019

(Nature) – Nick plunged himself into trying to find some relief for his son. As it turned out, researchers had identified a possible treatment. But the drug was being given to help infants survive a different condition — it wasn’t approved for AKU. And the path to approval was blocked by a considerable obstacle: the need for a full-scale randomized clinical trial. That’s expensive and difficult enough for medicines used to treat common diseases. It’s much harder for a condition that is almost unheard of.