Combination treatment of sarcoglycanopathies
US11298348B2 · kind B2 · utility
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Key dates
| Filing date | Jan 10, 2019 |
| Grant date | Apr 12, 2022 |
| Priority date | — |
| Expiry date | Jan 10, 2039 |
Classification
- Technology area (CPC A)Human Necessities
- CPC primaryA61P21/00
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
Sarcoglycanopathies are autosomal recessive diseases caused by mutations in the one of the genes coding for any sarcoglycans (SG). The inventors previously showed that the application of small molecules developed to rescue ΔF508-CFTR trafficking, and known as CFTR correctors, improved the maturation of several α-sarcoglycan mutants that were consequently rescued at the plasma membrane (WO 014086687). Now, the inventors show that some specific CFTR correctors provide additive and even synergic effect when administered in combination.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.