shylicine

Shylicine is an investigational small molecule drug being developed by Vanessa Research Holdings for the treatment of Microvillus Inclusion Disease (MVID). MVID is an extremely rare and life-threatening autosomal recessive genetic disorder characterized by severe, intractable secretory diarrhea and nutrient malabsorption starting in infancy. The condition is typically caused by mutations in the *MYO5B* gene, which leads to the loss of microvilli on the surface of enterocytes and the formation of characteristic inclusion bodies. Shylicine has been granted Orphan Drug Designation by the U.S. FDA for the treatment of MVID. The drug is intended to address the underlying pathophysiology of the disease to reduce the dependency on total parenteral nutrition and improve clinical outcomes for affected patients.

Brand names

Shylicine

Other names

Shylicine