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A trio against NGLY1 deficiency
October 2017
SHARING OPTIONS:

SAN DIEGO—Retrophin Inc., the National Center for Advancing Translational Sciences (NCATS) and NGLY1.org recently began a cooperative research and development agreement under which they will collaborate on research to identify potential treatments for NGLY1 deficiency. The rare genetic disease consists of developmental delays, seizure and an inability to produce tears, and has no approved therapeutic options. The organizations will seek to develop assays for small-molecule high-throughput screening in hopes of better understanding the biology of NGLY1 deficiency and identifying small molecules with potential as therapeutics. Retrophin will pursue continued development of compounds through the preclinical and clinical phases.
 
Dr. Bill Rote, senior vice president and head of research and development for Retrophin, said “This synergy between industry, public research and advocacy gives us a great opportunity to share resources with the common goal of making a difference in the lives of patients and families affected by NGLY1 deficiency, and Retrophin is proud to work alongside NCATS and NGLY1.org in this endeavor.”

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