Tetrahydrobiopterin synthesis and metabolism is impaired in dystrophin-deficient mdx mice and humans.
Angus LindsayBailey KempAlexie A LarsonCory W BaumannPreston M McCourtJohn HolmPeter KarachunskiDawn A LoweJames M ErvastiPublished in: Acta physiologica (Oxford, England) (2021)
These data demonstrate that impaired tetrahydrobiopterin synthesis is associated with dystrophin loss and treatment with tetrahydrobiopterin improves striated muscle histopathology and skeletal muscle function in mdx mice.