Ngly1 rats develop neurodegenerative phenotypes and pathological abnormalities

This Father Founded A Medical Research Startup To Save His Kid’s Life

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Gene therapy rescues disease phenotypes in NGLY1 rats.

The calculated U.S. incidence of NGLY1 Deficiency is ~ 12 individuals born per year.

Characterized and demonstrated the power of the NGLY1 Deficiency biomarker, GNA.