CN113329739B - 用于治疗scn1a脑病的靶向scn2a的反义寡核苷酸 - Google Patents

用于治疗scn1a脑病的靶向scn2a的反义寡核苷酸 Download PDF

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CN113329739B
CN113329739B CN201980068868.6A CN201980068868A CN113329739B CN 113329739 B CN113329739 B CN 113329739B CN 201980068868 A CN201980068868 A CN 201980068868A CN 113329739 B CN113329739 B CN 113329739B
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oligonucleotide
sugar
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CN113329739A (zh
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S.佩特鲁
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Luogong Co ltd
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CN201980068868.6A 2018-08-20 2019-08-20 用于治疗scn1a脑病的靶向scn2a的反义寡核苷酸 Active CN113329739B (zh)

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US201862765344P 2018-08-20 2018-08-20
US62/765,344 2018-08-20
PCT/US2019/047313 WO2020041348A1 (en) 2018-08-20 2019-08-20 Antisense oligonucleotides targeting scn2a for the treatment of scn1a encephalopathies

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CN113329739B true CN113329739B (zh) 2024-06-25

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AU (1) AU2019325255A1 (enExample)
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CN115867657A (zh) 2020-05-11 2023-03-28 斯托克制药公司 用于治疗疾患和疾病的opa1反义寡聚物
KR20230095056A (ko) * 2020-07-22 2023-06-28 더 플로레이 인스티튜트 오브 뉴로사이언스 앤드 멘탈 헬스 Scn2a에서 기능-상실 돌연변이와 연관된 장애를 치료하기 위한 조성물 및 방법
IL300258A (en) * 2020-08-07 2023-03-01 Ionis Pharmaceuticals Inc Compounds and methods for modulating SCN2A
EP4155402A1 (en) 2021-09-22 2023-03-29 Royal College of Surgeons in Ireland Modulation of microrna-335 for the treatment of sodium channelopathies
KR20250087733A (ko) * 2022-07-29 2025-06-16 스톡 테라퓨틱스, 인크. 병태 및 질환의 치료를 위한 화합물
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