First-Ever trial of sapropterin for rare smooth muscle syndrome begins
Disease control
Not yet recruiting
This study tests the drug sapropterin (Kuvan) in a single child with multisystem smooth muscle dysfunction syndrome (MSMDS), a rare genetic disorder with no approved treatment. The drug, already approved for another condition, aims to improve growth, blood pressure, and prevent b…
Phase: NA • Sponsor: Gregor Andelfinger • Aim: Disease control
Last updated Jun 27, 2026 12:30 UTC