Compounds and methods for modulating SMN2
US11299737B1 · kind B1 · utility
1Cited by
240References
32Claims
0Family size
Assignee
Inventor
Key dates
| Filing date | Jun 24, 2021 |
| Grant date | Apr 12, 2022 |
| Priority date | — |
| Expiry date | Jun 24, 2041 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12N2320/33
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
Provided are compounds, methods, and pharmaceutical compositions for modulating SMN2 RNA and/or protein in a cell or subject. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom of a neurodegenerative disorder. Such symptoms include reduced muscle strength; inability or reduced ability to sit upright, to stand, and/or walk; reduced neuromuscular activity; reduced electrical activity in one or more muscles; reduced respiration; inability or reduced ability to eat, drink, and/or breathe without assistance; loss of weight or reduced weight gain; and/or decreased survival.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.