Use of aminoglycoside analogs in the treatment of rett syndrome

a technology of aminoglycosides and rett syndrome, applied in the field of therapy, can solve the problems of reducing the suppression efficiency at subtoxic doses, compromising the clinical applicability of aminoglycosides of the gentamicin family,
US20170360817A1Inactive Publication Date: 2017-12-21ELOXX PHARM LTD
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Patent Information

Authority / Receiving Office
US · United States
Patent Type
Applications(United States)
Current Assignee / Owner
ELOXX PHARM LTD
Publication Date
2017-12-21
Estimated Expiration
Not applicable · inactive patent

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Abstract

Compounds, methods and uses of pseudo-trisaccharide aminoglycosides represented by the general formula I:or a pharmaceutically acceptable salt thereof, wherein R1 is selected from the group consisting of alkyl, cycloalkyl and aryl; and all other variables and features are as described in the specification, in the treatment of Rett syndrome are disclosed.
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Description

FIELD AND BACKGROUND OF THE INVENTION

[0001] The present invention, in some embodiments thereof, relates to therapy and, more particularly, but not exclusively, to compositions and methods utilizing aminoglycoside analogs in the treatment of Rett syndrome.

[0002] Rett syndrome (RTT, MIM 312750) is an X-linked postnatal neurodevelopmental disorder predominantly occurring in girls with a worldwide incidence of 1 / 10,000-15,000 female births. After normal development, during the first 6 to 18 months developmental stagnation and then regression occurs. During the phase of regression, purposeful hand use and language are lost while gross motor functions are relatively preserved. After the phase of regression the clinical picture remains stable for many years.

[0003] The major causative factor of RTT is deficiency of the X-linked methyl CpG-binding protein 2 (MeCP2) at Xq28, in which over 200 mutations have been identified so far in classical and atypical RTT patients. The majority of RTT causat...

Claims

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