COMPOSITIONS AND METHODS OF TREATING GALACTOSEMIA Russian patent published in 2024 - IPC A61K31/5025 A61K31/426 A61P3/00 

FIELD: medicine; pharmaceutics.

SUBSTANCE: group of inventions relates to methods of treating galactosemia and manifestations of galactosemia by administering to a subject in need thereof a therapeutically effective amount of an aldose reductase (AR) inhibitor, as well as to the use of AR inhibitors for the treatment of galactosemia and manifestations of galactosemia, where the AR inhibitor is a compound of formula III

or a pharmaceutically acceptable salt thereof, where R¹ is CO₂R²; R² is H, (C₁-C₆)-alkyl or (C₁-C₆)-aminoalkyl; X¹ is H; X² is H; Y is C=O; Z is

where A¹ is S; A² is N; A³ is O and R³-R⁶ are independently hydrogen, halogen or haloalkyl. Disclosed are methods of treating galactosemia or preventing complications associated with galactosemia; reducing the amount or level of galactite in a subject with galactosemia; treating cataracts in a subject with galactosemia; treating or preventing galactosemia-related cognitive or neurological impairment; as well as the use of an AR inhibitor, which is a compound of formula III, for the above purposes. Also disclosed are embodiments of said methods and applications using specific compounds of formula III.

EFFECT: use of said AR inhibitors can reduce or prevent accumulation of galactitol and the resulting pathology and complications associated with galactosemia.

56 cl, 8 dwg, 1 tbl, 4 ex