MULTISYSTEMIC SMOOTH MUSCLE DYSFUNCTION SYNDROME

Clinical trials for MULTISYSTEMIC SMOOTH MUSCLE DYSFUNCTION SYNDROME explained in plain language.

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Not yet recruiting 1

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…

Matched conditions: MULTISYSTEMIC SMOOTH MUSCLE DYSFUNCTION SYNDROME

Phase: NA • Sponsor: Gregor Andelfinger • Aim: Disease control

Last updated Jun 27, 2026 12:30 UTC

MULTISYSTEMIC SMOOTH MUSCLE DYSFUNCTION SYNDROME

Get alerted when new MULTISYSTEMIC SMOOTH MUSCLE DYSFUNCTION SYNDROME trials appear

First-Ever trial of sapropterin for rare smooth muscle syndrome begins