News from NORD

Collaborative Effort Is Underway to Screen Small Molecules for Therapeutic Potential for NGLY1 Deficiency

Publish date: December 7, 2017

 

A collaboration involving a patient organization (NGLY1.org), government (NIH National Center for Advancing Translational Sciences) and industry (Retrophin) is underway to screen hundreds of thousands of small molecules for their therapeutic potential in NGLY1 deficiency.

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