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20250177433. Compounds Meth (Ionis Pharmaceuticals, .)

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COMPOUNDS AND METHODS FOR REDUCING DMPK EXPRESSION

Abstract: provided are oligomeric compounds, methods, and pharmaceutical compositions for dmpk the amount or activity of dmpk rna in a cell or animal, and in certain instances reducing the amount of dmpk protein in a cell or animal. such oligomeric compounds, methods, and pharmaceutical compositions are useful to treat type 1 myotonic dystrophy.

Inventor(s): Frank Rigo, Eric E. Swayze, Paymaan Jafer-Nejad, Michael Oestergaard, Kar Yun Karen Ling, Susan M. Freier, Huynh-Hoa Bui

CPC Classification: A61K31/7125 (PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES (devices or methods specially adapted for bringing pharmaceutical products into particular physical or administering forms ; chemical aspects of, or use of materials for deodorisation of air, for disinfection or sterilisation, or for bandages, dressings, absorbent pads or surgical articles ; soap compositions ))

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