Genetics Program

Our innovative approach

Our team works closely with Children's researchers who are devoted to the study of genes that underlie inherited disorders and to finding new treatments and cures.

• Clinical trials are underway to test a drug developed at Children's to treat phenylketonuria, a disorder in which the body can't break down certain proteins.
• Children's Manton Center is the world's only center studying "orphan" diseases — those affecting fewer than 200,000 people, mostly children, in the United States.
• Researchers have found several genes linked to appetite and weight, paving the way for possible ways to prevent and treat obesity.