Phase III epalrestat trial is now enrolling.
Want to learn more about the PMM2-CDG clinical trial or our ongoing CDG research?
Would you like to become one of our research or clinical collaborators?
Based on the success of our foundational research with Perlara PBC and compassionate use trial of one patient in the US, the FDA has approved a prospective, single-center, randomized, double-blind, placebo-controlled clinical trial at Mayo Clinic for up to 40 PMM2-CDG children (ages 2-17).
"The improvement of PMM enzyme activity, and global glycosylation suggest epalrestat is a rational treatment target for PMM2-CDG and should be tested in a larger clinical trial."
Sorbitol is a severity biomarker for PMM2-CDG with therapeutic implications
Annals of Neurology
Word on the street
Congenital disorder of glycosylation – one
size does not fit all: a parent’s perspective
Groundbreaking biotech gets closer to possible treatment for kids with a rare disease
Check out the official government
clinical trial page
Maggie's Pearl wins
"Direct-to-Phase 2" SBIR award