ArticleAnnals of clinical and translational neurology2026

Re-Purposing Sapropterin (Kuvan) for ACTA2-Related Multisystemic Smooth Muscle Dysfunction Syndrome: A Translational Mechanistic and First-In-Human Therapeutic Report.

Moran Hausman-Kedem et al.PubMed ↗Full text ↗Publisher ↗

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