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Last Updated: December 16, 2025

Claims for Patent: 11,827,646

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Summary for Patent: 11,827,646

Title:	Compounds for treating spinal muscular atrophy
Abstract:	The present invention provides compounds of formula (I)wherein A, R1, R2 and R3 are as described herein, as well as pharmaceutically acceptable salts thereof. Further the present invention is concerned with the manufacture of the compounds of formula (I), pharmaceutical compositions comprising them and their use as medicaments.
Inventor(s):	Hasane Ratni, Luke Green, Marla L. Weetall, Nikolai A. Naryshkin
Assignee:	Hoffmann La Roche Inc , PTC Therapeutics Inc
Application Number:	US17/099,578
Patent Claims:	1. A method for the treatment of spinal muscular atrophy in a human in need thereof, said method comprising administering to said human a therapeutically effective amount of or a pharmaceutically acceptable salt thereof. 2. The method of claim 1, wherein the spinal muscular atrophy is Type 0 spinal muscular atrophy. 3. The method of claim 1, wherein the spinal muscular atrophy is Type 1 spinal muscular atrophy. 4. The method of claim 1, wherein the spinal muscular atrophy is Type 2 spinal muscular atrophy. 5. The method of claim 1, wherein the spinal muscular atrophy is Type 3 spinal muscular atrophy. 6. The method of claim 1, wherein the spinal muscular atrophy is Type 4 spinal muscular atrophy. 7. The method of claim 1, wherein the human is a human infant. 8. The method of claim 1, wherein the human is a human toddler. 9. The method of claim 1, wherein the human is a human child. 10. The method of claim 1, wherein the human is a human adult. 11. A method for the treatment of spinal muscular atrophy in a human in need thereof, said method comprising administering to said human a therapeutically effective amount of 12. A method for the treatment of spinal muscular atrophy in a human in need thereof, said method comprising administering to said human a pharmaceutical composition comprising or a pharmaceutically acceptable salt thereof, and one or more pharmaceutically acceptable excipients. 13. The method of claim 12, wherein the spinal muscular atrophy is Type 0 spinal muscular atrophy. 14. The method of claim 12, wherein the spinal muscular atrophy is Type 1 spinal muscular atrophy. 15. The method of claim 12, wherein the spinal muscular atrophy is Type 2 spinal muscular atrophy. 16. The method of claim 12, wherein the spinal muscular atrophy is Type 3 spinal muscular atrophy. 17. The method of claim 12, wherein the spinal muscular atrophy is Type 4 spinal muscular atrophy. 18. The method of claim 12, wherein the human is a human infant. 19. The method of claim 12, wherein the human is a human toddler. 20. The method of claim 12, wherein the human is a human child. 21. The method of claim 12, wherein the human is a human adult. 22. The method of claim 12, wherein the pharmaceutical composition is administered as an oral solution. 23. A method for the treatment of spinal muscular atrophy in a human in need thereof, said method comprising administering to said human a pharmaceutical composition comprising and one or more pharmaceutically acceptable excipients. 24. A method for the treatment of spinal muscular atrophy in a human in need thereof, said method comprising administering to said human a pharmaceutical composition comprising and one or more pharmaceutically acceptable excipients, wherein the pharmaceutical composition is administered as an oral solution.

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