Multisystemic Smooth Muscle Dysfunction Syndrome research hub

A Single Patient Study of Sapropterin for Multisystem Smooth Muscle Dysfunction Syndrome

This study is looking at a medicine called sapropterin (Kuvan) for children with a rare condition called Multisystem Smooth Muscle Dysfunction Syndrome (MSMDS). There's no approved treatment for MSMDS yet, so this study aims to see if Kuvan can help by gathering important information.