Invention Grant
- Patent Title: Methods for treating spinal muscular atrophy
- Patent Title (中): 治疗脊髓性肌萎缩的方法
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Application No.: US12994517Application Date: 2009-05-27
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Publication No.: US08633019B2Publication Date: 2014-01-21
- Inventor: Sergey V. Paushkin , Nikolai Naryshkin , Charles Romfo , Ellen Welch
- Applicant: Sergey V. Paushkin , Nikolai Naryshkin , Charles Romfo , Ellen Welch
- Applicant Address: US NJ South Plainfield
- Assignee: PTC Therapeutics, Inc.
- Current Assignee: PTC Therapeutics, Inc.
- Current Assignee Address: US NJ South Plainfield
- Agency: Jones Day
- International Application: PCT/US2009/003238 WO 20090527
- International Announcement: WO2009/151546 WO 20091217
- Main IPC: C12Q1/68
- IPC: C12Q1/68 ; A01N43/00 ; C12N15/00

Abstract:
The present invention provides nucleic acid constructs, methods for identifying and validating compounds that increase the inclusion of exon 7 of SMN2 into mRNA transcribed from the SMN2 gene, compounds and pharmaceutical compositions that increase levels of SMN protein produced from the SMN2 gene, and methods for use thereof in treating of SMA.
Public/Granted literature
- US20110086833A1 METHODS FOR TREATING SPINAL MUSCULAR ATROPHY Public/Granted day:2011-04-14
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