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公开(公告)号:US20230002324A1
公开(公告)日:2023-01-05
申请号:US17618428
申请日:2020-06-13
IPC分类号: C07D213/82 , C07D213/50 , C07D213/61 , C07D405/10 , C07D213/84 , C07D213/79 , C07D237/14 , C07D237/24 , C07D237/08 , C07D213/65
摘要: Disclosed herein are naphthoquinone derivative compounds of the formula shown below, compositions thereof, and methods of using such compounds and compositions for treating or suppressing oxidative stress disorders and/or neurodegenerative disorders and/or for inhibiting ferroptosis.
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公开(公告)号:US20220251098A1
公开(公告)日:2022-08-11
申请号:US17723163
申请日:2022-04-18
发明人: Matthew G. Woll , Lukiana Amedzo , Suresh Babu , Scott J. Barraza , Anuradha Bhattacharyya , Gary Mitchell Karp , Anthony R. Mazzotti , Jana Narasimhan , Jigar Patel , Anthony Turpoff , Zhenrong Xu
IPC分类号: C07D487/04 , C07D401/14 , C07D413/14 , C07D417/14 , C07D471/04 , C07D491/048 , C07D498/04 , C07D513/04 , C07D519/00
摘要: The present description relates to compounds, forms, and pharmaceutical compositions thereof and methods of using such compounds, forms, or compositions thereof for treating or ameliorating Huntington's disease. In particular, the present description relates to substituted bicyclic heteroaryl compounds of Formula (I), forms and pharmaceutical compositions thereof and methods of using such compounds, forms, or compositions thereof for treating or ameliorating Huntington's disease.
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公开(公告)号:US20220152223A1
公开(公告)日:2022-05-19
申请号:US17439140
申请日:2020-03-20
发明人: Antonio ARULANANDAM , Kevin NASH , Edwin WEEBER , Liangxian CAO , Min Jung KIM
摘要: One aspect described herein relates to a recombinant adeno-associated virus (rAAV) vector and a method for use thereof or treating Angelman Syndrome. Another aspect described herein is a UBE3A rAAV vector and method for use thereof for treating a UBE3A deficiency, e.g. Angelman syndrome, in humans.
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公开(公告)号:US20220151938A1
公开(公告)日:2022-05-19
申请号:US17588206
申请日:2022-01-28
发明人: Swathi Pinnamaneni
摘要: The present description relates to a tablet formulation of 7-(2,8-dimethylimidazo[1,2-b]pyridazin-6-yl)-5-fluoro-3-(piperidin-4-yl)benzo[e][1,2,4]triazine, a compound for use in treating Huntington's disease, and a method of making the same.
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公开(公告)号:US20220135586A1
公开(公告)日:2022-05-05
申请号:US17430511
申请日:2020-02-10
发明人: Nanjing ZHANG , Michael A. ARNOLD , Amal DAKKA , Gary Mitchell KARP , Tom Tuan LUONG , Jana NARASIMHAN , Nikolai A. NARYSHKIN , Jiashi WANG , Xiaoyan ZHANG
IPC分类号: C07D495/04 , A61P25/00
摘要: The present description relates' to compounds of formula (I) useful for improving pre-mRNA splicing in a cell. In particular, another aspect of the present description relates to substituted thieno[3,2-b]pyridine compounds, forms, and pharmaceutical compositions thereof and methods of use for treating or ameliorating familial dysautonomia.
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公开(公告)号:US11186559B2
公开(公告)日:2021-11-30
申请号:US16995754
申请日:2020-08-17
发明人: Peter Giannousis , Paul Mollard , Noah M. Benjamin , Jeffrey D. Butler , Olivier Dapremont , James B. Falabella
IPC分类号: C07D311/72
摘要: Provided herein are methods for making an alpha-tocotrienol enriched tocol mixture, such as from a plant or plant-derived material. Also provided herein are simulated moving bed purification methods for tocotrienol compounds such as alpha tocotrienol.
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公开(公告)号:US11083737B1
公开(公告)日:2021-08-10
申请号:US15607130
申请日:2017-05-26
IPC分类号: A61K31/58 , A61K45/06 , A61K9/00 , A61K9/10 , A61K9/20 , G06F19/00 , A61J1/05 , A61B5/00 , A61B5/22 , A61B5/08
摘要: The disclosure provides a deflazacort therapy comprising, administering to a subject suffering from Duchenne muscular dystrophy (DMD) a fixed dose of deflazacort. Also provided is a deflazacort therapy comprising, increasing the deflazacort dosage when the subject loses ambulation; or, increasing the dosage of deflazacort administered during concomitant administration of a CYP3A4 inducer; or, decreasing the dosage of deflazacort administered during concomitant administration of a CYP3A inhibitor.
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公开(公告)号:US20210236492A1
公开(公告)日:2021-08-05
申请号:US17267988
申请日:2019-08-17
发明人: Marla L. Weetall , Liangxian Cao , Thomas W. Davis , Melissa L. Dumble , Jaime A. Eberle-Singh , Kenneth P. Olive
IPC分类号: A61K31/506 , A61K31/7068 , A61K31/337 , A61P35/00
摘要: A method for treating pancreatic cancer in a subject in need thereof comprising, administering to the subject an effective amount of a tubulin polymerization inhibitor compound is described herein. More particularly, a method for treating pancreatic ductal adenocarcinoma in a subject in need thereof comprising, administering to the subject an effective amount of a substituted reverse pyrimidine tubulin polymerization inhibitor compound alone or in combination with other chemo-therapeutic agents is described herein.
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公开(公告)号:US20210147447A1
公开(公告)日:2021-05-20
申请号:US17099578
申请日:2020-11-16
IPC分类号: C07D519/00
摘要: The present invention provides compounds of formula (I) wherein A, R1, R2 and R3 are as described herein, as well as pharmaceutically acceptable salts thereof. Further the present invention is concerned with the manufacture of the compounds of formula (I), pharmaceutical compositions comprising them and their use as medicaments.
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公开(公告)号:US20210128494A1
公开(公告)日:2021-05-06
申请号:US16989753
申请日:2020-08-10
发明人: Guy M. MILLER , Viktoria Kheifets
摘要: Methods of treating or suppressing pervasive developmental disorders (PDDs) including; autistic disorder, Asperger's syndrome, childhood disintegrative disorder (CDD), Rett's disorder, and PDD-not otherwise specified (PDD-NOS) or attention deficit/hyperactivity disorder (ADHD) comprising administering to a subject in need thereof a therapeutically effective amount of one or more compounds as disclosed herein.
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