The standard treatment for some forms of congenital HI is diazoxide (right structure), a drug that controls insulin secretion by opening potassium channels in beta cells. However, this drug does not work in the most common types of HI, in which mutations prevent these potassium channels from forming.
A pilot study in adolescents and adults has found that an investigational drug (Exendin 9-39) (see below structure) shows promise as the first potential medical treatment for children with the severest type of congenital hyperinsulinism, a rare but potentially devastating disease in which gene mutations cause insulin levels to become dangerously high.
No comments:
Post a Comment