When Drug Development for Rare Disease Hit Setback, Parents Were Stung

A setback in development of a drug for the rare fatal disease known as NPC shows how steep the scientific and financial hurdles are for advancing promising drugs out of the lab and into patients.

When Drug Development For Rare Disease Hit Setback, Parents Were Stung

The Wall Street Journal – by Amy Dockser Marcus

A setback in development of a drug for the rare fatal disease known as NPC shows how steep the scientific and financial hurdles are for advancing promising drugs out of the lab and into patients.

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