The purpose of this study is to find out if an investigational drug called setmelanotide (RM-493) can help control body weight in people with certain genetic modifications in their genes that play a key role in the regulation of body weight.
The purpose of this study is to find out if the study drug, MAS825, is effective in preventing flares and safe for children with NLRC4-Gain of Function (GOF).
Our team at the Congenital Hyperinsulinism Center at CHOP is working on a research study to determine whether an experimental gene therapy product, DTX401, is safe and effective in people with glycogen storage disease type Ia (GSDIa)
Half of infants born with severe congenital heart disease go on to develop neurodevelopmental disorders, which may include cognitive, motor, social, and language impairments.
