-
公开(公告)号:CO2024017547A2
公开(公告)日:2025-03-27
申请号:CO2024017547
申请日:2024-12-20
Applicant: PTC THERAPEUTICS INC
Inventor: BHATTACHARYYA ANURADHA , CHEN GUANGMING , JIANG YAO , NARASIMHAN JANA , WOLL MATTHEW G , ZHANG NANJING , ZHANG XIAOYAN , ARNOLD MICHAEL A , ANNAND JAMES R , ARACHCHILAGE GAYAN MIRIHANA , CHIU JACKIE , DAHIYA GAURAV , KIZHAKKAYIL MANGADAN ARUN RAJ , MORRILL CHRISTIE , MOTIKA STEPHEN E
IPC: A61K31/4365 , A61K31/517 , A61K31/519 , C07D495/04
Abstract: Un aspecto de la presente descripción se refiere a compuestos útiles para mejorar el corte y empalme de pre ARNm en una célula. En particular, otro aspecto de la presente descripción se refiere a compuestos heteroarilo sustituidos, formas y composiciones farmacéuticas de los mismos y métodos de uso para tratar o mejorar la ataxia espinocerebelosa tipo 3 (SCA3), también conocida como enfermedad de Machado-Joseph (MJD).
-
公开(公告)号:MX2011010482A
公开(公告)日:2012-01-30
申请号:MX2011010482
申请日:2010-04-05
Applicant: PTC THERAPEUTICS INC
Inventor: NJOROGE F GEORGE , LENNOX WILLIAM JOSEPH , TAKASUGI JAMES , REN HONGYU , KARP GARY MITCHELL , LI CHUNSHI , ZHANG XIAOYAN , ZHU JIN , LIU RONGGANG , MACCOSS MALCOLM , NOMEIR AMIN , CHEN GUANGMING , HUANG SONG XIAO , KAKARLA RAMESH , LIU YALEI , MORRILL CHRISTIE , PAGET STEVEN D , SMITH SEAN W , TURPOFF ANTHONY A , ZHANG NANJING
IPC: C07D401/04 , A61K31/443 , A61K31/4433 , A61K31/4436 , A61K31/4439 , A61K31/444 , A61K31/506 , A61P31/12 , C07D401/14 , C07D403/04 , C07D405/14 , C07D409/14 , C07D413/14 , C07D417/14
Abstract: La presente invención está dirigida a compuestos y formas y composiciones farmacéuticas de los mismos, útiles para el tratamiento de una infección viral, o para afectar la actividad viral modulando la replicación viral.
-
公开(公告)号:CO2022008195A2
公开(公告)日:2022-07-29
申请号:CO2022008195
申请日:2022-06-10
Applicant: PTC THERAPEUTICS INC
Inventor: KARP GARY MITCHELL , NARASIMHAN JANA , TURPOFF ANTHONY , ZHANG NANJING , DAKKA AMAL , WANG JIASHI , ZHANG XIAOYAN , MORRILL CHRISTIE , LUONG TOM TUAN , ARNOLD MICHAEL A , NARYSHKIN NIKOLAI A
IPC: A61P25/00 , A61K31/519 , C07D495/04
Abstract: La presente descripción se refiere a compuestos útiles para mejorar el corte y empalme de pre-ARNm en una célula. En particular, otro aspecto de la presente descripción se refiere a compuestos tieno[3,2-d]pirimidina sustituidos, formas y composiciones farmacéuticas de los mismos y a métodos de uso para tratar o mejorar la disautonomía familiar.
-
公开(公告)号:BR112021022624A2
公开(公告)日:2022-01-04
申请号:BR112021022624
申请日:2020-05-12
Applicant: PTC THERAPEUTICS INC
Inventor: TURPOFF ANTHONY , ANTHONY R MAZZOTTI , BHATTACHARYYA ANURADHA , MORRILL CHRISTIE , WANG GANG , GARY MITCHELL KARP , REN HONGYU , NARASIMHAN JANA , JIGAR S PATEL , AMEDZO LUKIANA , MATTHEW G WOLL , MD RAUFUL ALAM , MICHAEL A ARNOLD , SYDORENKO NADIYA , ZHANG NANJING , NATHANIEL T KENTON , NICHOLAS W MSZAR , BABU SURESH , TOM TUAN LUONG , ZHANG XIAOYAN , MOON YOUNG-CHOON
IPC: A61K31/53 , A61K31/501 , A61P25/28 , C07D401/14 , C07D413/14 , C07D417/14 , C07D487/04
Abstract: compostos para o tratamento da doença de huntington. a presente invenção refere-se a compostos, formas, e composições farmacêuticas dos mesmos e métodos de uso de tais compostos, formas, ou composições dos mesmos para tratar ou melhorar a doença de huntington. em particular, a presente descrição refere-se a compostos heteroarila monocíclicos substituídos de fórmula (i), fórmula (ii), ou fórmula (iii), formas e composições farmacêuticas dos mesmos e métodos de uso de tais compostos, formas, ou composições dos mesmos para tratar ou melhorar a doença de huntington.
-
公开(公告)号:SG11202112504YA
公开(公告)日:2021-12-30
申请号:SG11202112504Y
申请日:2020-05-12
Applicant: PTC THERAPEUTICS INC
Inventor: SYDORENKO NADIYA , ALAM MD RAUFUL , AMEDZO LUKIANA , ARNOLD MICHAEL A , BABU SURESH , BHATTACHARYYA ANURADHA , KARP GARY MITCHELL , KENTON NATHANIEL T , LUONG TOM TUAN , MAZZOTTI ANTHONY R , MOON YOUNG-CHOON , MORRILL CHRISTIE , NARASIMHAN JANA , MSZAR NICHOLAS WALTER , PATEL JIGAR S , REN HONGYU , TURPOFF ANTHONY , WANG GANG , WOLL MATTHEW G , ZHANG NANJING , ZHANG XIAOYAN
IPC: C07D401/14 , A61K31/501 , A61K31/53 , A61P25/28 , C07D413/14 , C07D417/14 , C07D487/04
Abstract: 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 monocyclic heteroaryl compounds of Formula (I), Formula (II), or Formula (III), forms and pharmaceutical compositions thereof and methods of using such compounds, forms, or compositions thereof for treating or ameliorating Huntington's disease.
-
公开(公告)号:SG175047A1
公开(公告)日:2011-11-28
申请号:SG2011072162
申请日:2010-04-05
Applicant: SCHERING CORP , PTC THERAPEUTICS INC
Inventor: MACCOSS MALCOLM , NJOROGE F GEORGE , NOMEIR AMIN , CHEN GUANGMING , HUANG SONG XIAO , KAKARLA RAMESH , KARP GARY MITCHELL , LENNOX WILLIAM JOSEPH , LI CHUNSHI , LIU RONGGANG , LIU YALEI , MORRILL CHRISTIE , PAGET STEVEN D , SMITH SEAN W , TAKASUGI JAMES , TURPOFF ANTHONY A , REN HONGYU , ZHANG NANJING , ZHANG XIAOYAN , ZHU JIN
Abstract: The present invention is directed to compounds and forms and pharmaceutical compositions thereof useful for treating a viral infection, or for affecting viral activity by modulating viral replication.
-
公开(公告)号:CA2757640A1
公开(公告)日:2010-10-14
申请号:CA2757640
申请日:2010-04-05
Applicant: PTC THERAPEUTICS INC
Inventor: MACCOSS MALCOLM , NJOROGE F GEORGE , NOMEIR AMIN , CHEN GUANGMING , HUANG SONG XIAO , KAKARLA RAMESH , KARP GARY MITCHELL , LENNOX WILLIAM JOSEPH , LI CHUNSHI , LIU RONGGANG , LIU YALEI , MORRILL CHRISTIE , PAGET STEVEN D , SMITH SEAN W , TAKASUGI JAMES , TURPOFF ANTHONY A , REN HONGYU , ZHANG NANJING , ZHANG XIAOYAN , ZHU JIN
IPC: C07D401/04 , A61K31/443 , A61K31/4433 , A61K31/4436 , A61K31/4439 , A61K31/444 , A61K31/506 , A61P31/12 , C07D401/14 , C07D403/04 , C07D405/14 , C07D409/14 , C07D413/14 , C07D417/14
Abstract: The present invention is directed to compounds and forms and pharmaceutical compositions thereof useful for treating a viral infection, or for affecting viral activity by modulating viral replication.
-
公开(公告)号:MA71255A
公开(公告)日:2025-04-30
申请号:MA71255
申请日:2023-06-20
Applicant: PTC THERAPEUTICS INC
Inventor: NARASIMHAN JANA , ZHANG NANJING , ZHANG XIAOYAN , BHATTACHARYYA ANURADHA , CHEN GUANGMING , JIANG YAO , WOLL MATTHEW G , ARNOLD MICHAEL A , ANNAND JAMES R , ARACHCHILAGE GAYAN MIRIHANA , CHIU JACKIE , DAHIYA GAURAV , KIZHAKKAYIL MANGADAN ARUN RAJ , MORRILL CHRISTIE , MOTIKA STEPHEN E
IPC: A61K31/4365 , A61K31/517 , A61K31/519 , C07D495/04
-
公开(公告)号:AU2020402717A1
公开(公告)日:2022-06-23
申请号:AU2020402717
申请日:2020-12-07
Applicant: PTC THERAPEUTICS INC
Inventor: ZHANG NANJING , ARNOLD MICHAEL A , DAKKA AMAL , KARP GARY MITCHELL , LUONG TOM TUAN , MORRILL CHRISTIE , NARASIMHAN JANA , NARYSHKIN NIKOLAI A , TURPOFF ANTHONY , WANG JIASHI , ZHANG XIAOYAN
IPC: A61P25/00 , A61K31/519 , C07D495/04
Abstract: The present description relates to compounds useful for improving pre-mRNA splicing in a cell. In particular, another aspect of the present description relates to substituted thieno [3,2-d]pyrimidine compounds, forms, and pharmaceutical compositions thereof and methods of use for treating or ameliorating familial dysautonomia.
-
公开(公告)号:AU2020277027A1
公开(公告)日:2021-12-09
申请号:AU2020277027
申请日:2020-05-12
Applicant: PTC THERAPEUTICS INC
Inventor: SYDORENKO NADIYA , ALAM MD RAUFUL , AMEDZO LUKIANA , ARNOLD MICHAEL A , BABU SURESH , BHATTACHARYYA ANURADHA , KARP GARY MITCHELL , KENTON NATHANIEL T , LUONG TOM TUAN , MAZZOTTI ANTHONY R , MOON YOUNG-CHOON , MORRILL CHRISTIE , MSZAR NICHOLAS WALTER , NARASIMHAN JANA , PATEL JIGAR S , REN HONGYU , TURPOFF ANTHONY , WANG GANG , WOLL MATTHEW G , ZHANG NANJING , ZHANG XIAOYAN
IPC: C07D401/14 , A61K31/501 , A61K31/53 , A61P25/28 , C07D413/14 , C07D417/14 , C07D487/04
Abstract: 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 monocyclic heteroaryl compounds of Formula (I), Formula (II), or Formula (III), forms and pharmaceutical compositions thereof and methods of using such compounds, forms, or compositions thereof for treating or ameliorating Huntington's disease.
-
-
-
-
-
-
-
-
-