Palvella - Model QTORIN - Gorlin Syndrome

Gorlin syndrome, also known as nevoid basal cell carcinoma syndrome, is a serious rare life-long genetic disease caused by a mutation in the PTCH1 gene leading to abherrant hedgehog signaling. Recurrent basal cell carcinomas (BCCs) are a hallmark of this disease.  Individuals with Gorlin syndrome can develop hundreds or even thousands of BCCs, oftentimes beginning in childhood. BCCs are a malignant skin cancer requiring repeated and potentially disfiguring surgical removal for patients with Gorlin syndrome.  Gorlin syndrome affects an estimated 11,000 people in the United States.

There are no FDA approved therapies for Gorlin syndrome.