Perlara

South San Francisco, California, United States

The development of the first platform therapy for a group of related genetic inborn errors of metabolism known as congenital disorders of glycosylation (CDG) has been achieved. The cure candidate, Epalrestat, an aldose reductase inhibitor approved in Japan in 1992 for neuropathy in adults with diabetes, targets the root cause of PMM2-CDG, the most common CDG affecting over 1000 patients worldwide. Building upon foundational research and successful individual treatments, a pivotal Phase 3 trial has been initiated for up to 40 PMM2-CDG children (ages 2-17) at a single clinical trial site at the Mayo Clinic. The clinical protocol incorporates telemedicine visits. Additionally, research into treatments for other CDGs is underway. The success of this approach relies on the scientific acumen and leadership of Perlara Cure Guides™.