Laron Syndrome : Treating Growth Disorders | Cincinnati Children’s

Laron syndrome is a condition that occurs when the body is unable to utilize growth hormone. It is primarily characterized by short stature.  It is often caused by mutations in the GHR gene and is inherited in an autosomal recessive manner. Treatment is focused on improving growth and generally includes injections of insulin-like growth factor 1 (IGF-1).

Type 1 involves a defect in the growth hormone receptor gene which prevents the hormone from binding and being used. Hence there are high levels of free growth hormone in the plasma. Type 2 involves a problem with the processing of the growth hormone once it has been bound properly to the cell surface.

Three-year-old Brayden Newbold is from the Bahamas. His parents brought him to Cincinnati Children’s Hospital Medical Center for treatment when they noticed he wasn’t growing as expected.Dr. Philippe Backeljauw diagnosed him with Laron syndrome, a growth disorder that causes small stature. In the 1990’s, researchers at Cincinnati Children’s conducted a study for a drug called Increlex for treatment of patients with Laron syndrome. Patients from all over the world took part in the study. The Food and Drug Administration approved the drug for patients in 2005. Brayden Newbold has grown 3 1/2 inches since taking Increlex injections over the last 9 months. Brayden continues receiving treatment at Cincinnati Children’s Growth Center in the division of Endocrinology.