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公开(公告)号：US10905709B2
公开(公告)日：2021-02-02
申请号：US15754782
申请日：2016-08-26
发明人： Marco A. Passini , Gunnar J. Hanson
IPC分类号： A61K31/712 , C12N15/11 , A61K31/7125 , A61P25/14 , A61P21/00
摘要： The present disclosure relates to modified antisense oligomers and related compositions and methods for increasing the expression of functional SMN protein and methods for treating spinal muscular atrophy and relates to inducing inclusion of exon 7 in SMN2 mRNA.
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公开(公告)号：US20210009639A1
公开(公告)日：2021-01-14
申请号：US16924745
申请日：2020-07-09
摘要： Described herein are isolated peptides, compositions comprising the same, and methods of using such peptides or compositions in the treatment of depression, central nervous system disorders, and neurodevelopmental disorders.
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公开(公告)号：US10857137B2
公开(公告)日：2020-12-08
申请号：US16870706
申请日：2020-05-08
IPC分类号： A61K31/4375 , A61P25/14 , A61K31/4745 , A61K9/00 , A61K9/48 , A61K31/4525 , A61K31/4709
摘要： Provided are methods of administering a vesicular monoamine transport 2 (VMAT2) inhibitor chosen from valbenazine and (+)-α-3-isobutyl-9,10-dimethoxy-1,3,4,6,7,11b-hexahydro-2H-pyrido[2,1-a]isoquinolin-2-ol, or a pharmaceutically acceptable salt and/or isotopic variant thereof, to a patient in need thereof wherein the patient is a CYP2D6 poor metabolizer.
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公开(公告)号：US20200268725A1
公开(公告)日：2020-08-27
申请号：US16870706
申请日：2020-05-08
IPC分类号： A61K31/4375 , A61K31/4745 , A61P25/14
摘要： Provided are methods of administering a vesicular monoamine transport 2 (VMAT2) inhibitor chosen from valbenazine and (+)-α-3-isobutyl-9,10-dimethoxy-1, 3,4, 6,7,11 b-hexahydro-2H-pyrido[2,1-a]isoquinolin-2-ol, or a pharmaceutically acceptable salt and/or isotopic variant thereof, to a patient in need thereof wherein the patient is a CYP2D6 poor metabolizer.
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公开(公告)号：US10710976B2
公开(公告)日：2020-07-14
申请号：US16272788
申请日：2019-02-11
发明人： Reza Mazhari , Djelila Mezaache , Blake M. Paterson , James Vornov , Rachel M. Garner , Todd Nelson
IPC分类号： C07D401/12 , A61P9/10 , A61P9/00 , A61P5/24 , A61P43/00 , A61P29/02 , A61P27/16 , A61P27/06 , A61P25/28 , A61P25/24 , A61P25/18 , A61P25/16 , A61P25/14 , A61P25/08 , A61P25/04 , A61P25/00 , A61P17/02 , A61P15/00 , A61P11/14
摘要： The disclosed subject matter provides certain polymorphic forms of Compound (I) as well as pharmaceutical compositions comprising Compound (I) or such polymorphic forms, and methods of using or making such compounds and pharmaceutical compositions. It has now been discovered that Compound (I) can exist in multiple crystalline forms (polymorphs). One particular crystalline form, Form II, has been found to be more thermodynamically stable and, thus, likely more suitable for bulk preparation and handling than other polymorphic forms. Efficient and economic methods have been developed to prepare Compound (I) and Form II in high purity on a large scale. In animal studies, Form II has demonstrated safety and efficacy in treating depressive disorders and, when micronized, improved absorption compared to non-micronized Form II.
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公开(公告)号：US10703811B2
公开(公告)日：2020-07-07
申请号：US16026482
申请日：2018-07-03
申请人： University of Miami
发明人： Robert W. Keane , W. Dalton Dietrich , Juan Pablo De Rivero Vaccari , Helen M. Bramlett , Roberta Brambilla
IPC分类号： C07K16/24 , A61P25/28 , A61P29/00 , C07K16/18 , A61P11/00 , A61P25/00 , A61K31/727 , A61K39/395 , A61P25/14 , A61P37/06 , A61K39/00
摘要： The compositions and methods described herein include agents that inhibit inflammasome signaling in the mammal such as antibodies directed against inflammasome components used alone or in combination with extracellular vesicle uptake inhibitor(s). Also described herein are compositions and methods of use thereof for treating multiple sclerosis.
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公开(公告)号：US20200179355A1
公开(公告)日：2020-06-11
申请号：US16789564
申请日：2020-02-13
发明人： Michal GEVA , Ralph LAUFER , Michael HAYDEN , Neta ZACH
IPC分类号： A61K31/44 , A61K9/00 , A61K31/426 , A61K31/4152 , A61K31/49 , A61K31/222 , A61K31/185 , A61K31/4748 , A61P25/14
摘要： Provided herein is a method for treating a human subject afflicted with ALS by administering to the subject a therapeutically effective amount of pridopidine as monotherapy or together with riluzole, edaravone, combination of dextromethorphan/quinidine, sodium phenylbutyrate (PB), tauroursodeoxycholic acid or combination of sodium phenylbutyrate (PB)/tauroursodeoxycholic acid (i.e. AMX0035) as combination or add-on therapy.
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58.发明申请公开(公告)号：US20200171042A1
公开(公告)日：2020-06-04
申请号：US16709988
申请日：2019-12-11
发明人： Delphine Charvin , François Conquet
IPC分类号： A61K31/5377 , C07D495/04 , A61P25/14 , A61P25/16 , A61K9/00 , A61K31/198 , A61K45/06
摘要： The present invention provides a chromone oxime derivative of formula (I), which is a modulator of nervous system receptors sensitive to the neuroexcitatory amino acid glutamate and presents an advantageously high brain exposure upon oral administration, for the treatment or prevention of levodopa-induced dyskinesia. The present invention also provides an improved therapy of Parkinson's disease, using the chromone oxime derivative of formula (I) in combination with levodopa.
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公开(公告)号：US20200155624A1
公开(公告)日：2020-05-21
申请号：US16611041
申请日：2018-05-04
发明人： Dinah Wen-Yee Sah , Fen Chen , Pengcheng Zhou , Xin Wang , Yanqun Shu , Jinzhao Hou
IPC分类号： A61K35/76 , C12N7/00 , A61P25/14 , C12N15/113 , C12N15/86
摘要： The present invention relates to adeno-associated viral (AAV) particles encoding siRNA molecules and methods for treating Huntington's Disease (HD).
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公开(公告)号：US10647761B2
公开(公告)日：2020-05-12
申请号：US16358935
申请日：2019-03-20
发明人： Klaus Kaluza , Olaf Mundigl , Thomas Kremer , Markus Britschgi , Sylwia Huber
摘要： The invention provides anti-human alpha-synuclein antibodies and methods of using the same.
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