Breakthrough treatment for CDG, a rare disease affecting over 1000 patients worldwide


Maggie’s Pearl is a biotech joint venture founded by Perlara and Maggie’s Cure on a mission to develop the first platform therapy for a group of related genetic inborn errors of metabolism called congenital disorders of glycosylation (CDG). Epalrestat, an aldose reductase inhibitor approved in Japan in 1992 for neuropathy in adults with diabetes, also targets the root cause of PMM2-CDG, the most common CDG affecting over 1000 patients worldwide.

Join us in our quest to cure CDG


Based on the success of our foundational research and Maggie's n-of-1 treatment, we're preparing to enroll for a clinical trial for up to 30 PMM2-CDG children (ages 2-17). There will be a single clinical trial site, and the clinical protocol includes telemedicine visits. Research into treatments for other CDGs is planned.


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