JP2016537341A5 - - Google Patents

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JP2016537341A5
JP2016537341A5 JP2016528904A JP2016528904A JP2016537341A5 JP 2016537341 A5 JP2016537341 A5 JP 2016537341A5 JP 2016528904 A JP2016528904 A JP 2016528904A JP 2016528904 A JP2016528904 A JP 2016528904A JP 2016537341 A5 JP2016537341 A5 JP 2016537341A5
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Priority claimed from PCT/US2014/064987 external-priority patent/WO2015070212A1/en
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JP2016528904A 2013-11-11 2014-11-11 ハンチントン病を処置するための方法および組成物 Pending JP2016537341A (ja)

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US201361902704P 2013-11-11 2013-11-11
US61/902,704 2013-11-11
PCT/US2014/064987 WO2015070212A1 (en) 2013-11-11 2014-11-11 Methods and compositions for treating huntington's disease

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JP2016537341A JP2016537341A (ja) 2016-12-01
JP2016537341A5 true JP2016537341A5 (enExample) 2017-10-12

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JP2016528904A Pending JP2016537341A (ja) 2013-11-11 2014-11-11 ハンチントン病を処置するための方法および組成物
JP2019067000A Pending JP2019116502A (ja) 2013-11-11 2019-03-29 ハンチントン病を処置するための方法および組成物

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US (1) US10369201B2 (enExample)
EP (1) EP3068905A4 (enExample)
JP (2) JP2016537341A (enExample)
KR (1) KR102431079B1 (enExample)
CN (1) CN105934524A (enExample)
AU (1) AU2014346424B2 (enExample)
BR (1) BR112016010175A2 (enExample)
CA (1) CA2929179A1 (enExample)
MX (1) MX391904B (enExample)
NZ (1) NZ719477A (enExample)
RU (1) RU2693891C1 (enExample)
WO (1) WO2015070212A1 (enExample)

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