METHODS OF TREATING CREATINE TRANSPORTER DEFICIENCY
Disclosed are methods of treating creatine transporter deficiency, comprising administering to a mammal in need thereof a therapeutically effective amount of a compound that increases transport of a substrate by a mutant or wild-type creatine transporter. Also disclosed are methods of increasing transport of guanidinoacetic acid or a salt thereof across the blood-brain barrier of a mammal, and methods of decreasing accumulation or the concentration of guanidinoacetic acid or a salt thereof in a mammalian cell.
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Paragraph 0611-0612
(2021/10/02)
SMALL MOLECULES TARGETING MUTANT MAMMALIAN PROTEINS
Disclosed are compounds, compositions, and methods useful for treating or preventing a disease or disorder associated with a mutation in a protein.
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Paragraph 0235; 0236; 0255
(2021/10/02)
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