CN114302727A - 治疗或预防脊髓性肌萎缩的方法 - Google Patents

治疗或预防脊髓性肌萎缩的方法 Download PDF

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CN114302727A
CN114302727A CN202080049461.1A CN202080049461A CN114302727A CN 114302727 A CN114302727 A CN 114302727A CN 202080049461 A CN202080049461 A CN 202080049461A CN 114302727 A CN114302727 A CN 114302727A
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antisense compound
salt
loading dose
dose
human subject
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K.S.洛夫迪
F.J.白
L.伊斯特
W.R.法韦尔
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Biogen MA Inc
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Biogen MA Inc
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CN202080049461.1A 2019-07-19 2020-07-16 治疗或预防脊髓性肌萎缩的方法 Pending CN114302727A (zh)

Applications Claiming Priority (3)

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US201962876360P 2019-07-19 2019-07-19
US62/876,360 2019-07-19
PCT/US2020/042312 WO2021016032A1 (en) 2019-07-19 2020-07-16 Methods of treating or preventing spinal muscular atrophy

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Families Citing this family (4)

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Publication number Priority date Publication date Assignee Title
EP3797780B1 (en) 2014-04-17 2022-09-14 Biogen MA Inc. Compositions and methods for modulation of smn2 splicing in a subject
US10436802B2 (en) 2014-09-12 2019-10-08 Biogen Ma Inc. Methods for treating spinal muscular atrophy
PT3999643T (pt) 2019-07-19 2023-12-11 Biogen Ma Inc Métodos de tratamento da atrofia muscular espinhal
WO2024138018A1 (en) * 2022-12-23 2024-06-27 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Treatment of gemin5-mediated disorders

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2008157753A1 (en) * 2007-06-21 2008-12-24 Sma Foundation Methods of treatment for spinal muscular atrophy
US20140113956A1 (en) * 2011-05-25 2014-04-24 Universite Paris Descartes Erk inhibitors for use in treating spinal muscular atrophy
US20160002627A1 (en) * 2013-01-09 2016-01-07 Isis Pharmaceuticals. Inc. Compositions and methods for modulation of smn2 splicing in a subject

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3470072A1 (en) * 2005-06-23 2019-04-17 Biogen MA Inc. Compositions and methods for modulation of smn2 splicing
ES2699827T3 (es) 2009-06-17 2019-02-13 Biogen Ma Inc Composiciones y métodos para la modulación de corte y empalme de SMN2 en un sujeto
WO2011032109A1 (en) 2009-09-11 2011-03-17 Sma Foundation Biomarkers for spinal muscular atrophy
US10436802B2 (en) 2014-09-12 2019-10-08 Biogen Ma Inc. Methods for treating spinal muscular atrophy
EP3484524B1 (en) * 2016-07-15 2022-11-09 Ionis Pharmaceuticals, Inc. Compounds and methods for modulation of smn2
TW201927241A (zh) 2017-12-21 2019-07-16 瑞士商赫孚孟拉羅股份公司 用於肌肉失能之數位生物標記
CN119679944A (zh) 2018-01-25 2025-03-25 渤健马萨诸塞州股份有限公司 治疗脊髓性肌萎缩症的方法
IT201800004359A1 (it) 2018-04-10 2019-10-10 IDENTIFICAZIONE DI miRNA MUSCOLARI COME BIOMARCATORI E TRATTAMENTO CO-ADIUVANTE PER L’ATROFIA MUSCOLARE SPINALE
US20210308281A1 (en) * 2018-08-15 2021-10-07 Biogen Ma Inc. Combination therapy for spinal muscular atrophy
PT3999643T (pt) 2019-07-19 2023-12-11 Biogen Ma Inc Métodos de tratamento da atrofia muscular espinhal

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2008157753A1 (en) * 2007-06-21 2008-12-24 Sma Foundation Methods of treatment for spinal muscular atrophy
US20140113956A1 (en) * 2011-05-25 2014-04-24 Universite Paris Descartes Erk inhibitors for use in treating spinal muscular atrophy
US20160002627A1 (en) * 2013-01-09 2016-01-07 Isis Pharmaceuticals. Inc. Compositions and methods for modulation of smn2 splicing in a subject

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
RICHARD S FINKEL ET AL.: "Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study", LANCET, vol. 388, pages 3017 - 3026, XP029849269, DOI: 10.1016/S0140-6736(16)31408-8 *

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US20220325282A1 (en) 2022-10-13
EP4335503A3 (en) 2024-06-05
JOP20220007A1 (ar) 2023-01-30
HRP20231515T1 (hr) 2024-03-01
TW202116331A (zh) 2021-05-01
US20240318182A1 (en) 2024-09-26
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FI3999643T3 (fi) 2023-11-30
BR112022000870A2 (pt) 2022-03-22
JOP20250103A1 (ar) 2025-05-05
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EP3999643B1 (en) 2023-11-22
DK3999643T5 (da) 2024-08-05
JP2025096486A (ja) 2025-06-26
IL289745A (en) 2022-03-01
LT3999643T (lt) 2023-12-11
SMT202300442T1 (it) 2024-01-10
AU2020316997A1 (en) 2022-03-10
CL2021003602A1 (es) 2022-10-14
MD3999643T2 (ro) 2024-02-29
AR119430A1 (es) 2021-12-15
DK3999643T3 (da) 2023-12-04
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JP2022540993A (ja) 2022-09-21
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