A new study explores the repurposing of Sapropterin (Kuvan) for treating ACTA2-related multisystemic smooth muscle dysfunction syndrome. This first-in-human therapeutic report provides insights into the drug's potential mechanisms and applications.
re purposing sapropterin kuvan for acta2 related multisystemic smooth muscle dysfunction syndrome a translational mechanistic and first in human therapeutic report
Original title: “Re-Purposing Sapropterin (Kuvan) for ACTA2-Related Multisystemic Smooth Muscle Dysfunction Syndrome: A Translational Mechanistic and First-In-Human Therapeutic Report.”