Potent transthyretin (ttr) stabilization in ttr amyloidosis patients receiving acoramidis

A method of treating transthyretin (TTR) amyloidosis in a subject in need thereof, the method comprising administering to a subject in need thereof a therapeutically effective amount of Compound 1, having the formula:

• or a pharmaceutically acceptable salt thereof, wherein said therapeutically effective amount is a total daily dosage of about 10 milligrams (mg) to about 2,000 mg of Compound 1 in HCl salt form or an equivalent amount of Compound 1 in free base form or in a different salt form, and
• wherein the TTR amyloidosis in said subject is characterized by a TTR protein comprising a mutation selected from the group consisting of:
• a glycine to serine mutation at position 6 (G6S),
• an alanine to serine mutation at position 25 (A25S),
• an alanine to aspartic acid mutation at position 36 (A36D),
• a glutamic acid to aspartic acid mutation at position 42 (E42D),
• a serine to arginine mutation at position 50 (S50R),
• an isoleucine to leucine mutation at position 68 (168L),
• a glutamic acid to glutamine mutation at position 89 (E89Q),
• a glutamic acid to glutamine mutation at position 92 (E92Q),
• a valine to leucine mutation at position 94 (V94L), and
• an alanine to serine mutation at position 97 (A97S).