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.: US13058653Application Date: 2009-08-13
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Publication No.: US08986935B2Publication Date: 2015-03-24
- Inventor: Sergey V. Paushkin , Nikolai A. Naryshkin , Ellen Welch
- Applicant: Sergey V. Paushkin , Nikolai A. Naryshkin , 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/US2009/004625 WO 20090813
- International Announcement: WO2010/019236 WO 20100218
- Main IPC: C12Q1/68
- IPC: C12Q1/68 ; G01N33/567 ; C12Q1/02
Abstract:
Described herein are methods for the identification or validation of compounds capable of causing ribosomal frameshifting and the use of the compounds identified by the methods described herein to produce a stabilized SMNΔEx7 protein and treat Spinal Muscular Atrophy.
Public/Granted literature
- US20110172284A1 METHODS FOR TREATING SPINAL MUSCULAR ATROPHY Public/Granted day:2011-07-14
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