Methods for treating spinal muscular atrophy
US10436802B2 · kind B2 · utility
9Cited by
25References
20Claims
0Family size
Assignee
Inventors
Key dates
| Filing date | Sep 11, 2015 |
| Grant date | Oct 8, 2019 |
| Priority date | — |
| Expiry date | Sep 11, 2035 |
Classification
- Technology area (CPC G)Physics
- CPC primaryG01N2800/52
- WIPO fieldMeasurement
- WIPO sectorInstruments
Abstract
Disclosed herein are compounds, compositions and methods for modulating splicing of SMN2 mRNA in a subject. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders, including spinal muscular atrophy. Also provided are kits for detecting the amount of SMN protein in a sample of cerebrospinal fluid.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.