公开(公告)号：AU2017204248A1

公开(公告)日：2017-07-13

申请号：AU2017204248

申请日：2017-06-22

Applicant: PTC THERAPEUTICS INC ,  F HOFFMAN-LA ROCHE AG

Inventor： QI HONGYAN ,  CHOI SOONGYA ,  DAKKA AMAL ,  KARP GARY MITCHELL ,  NARASIMHAN JANA ,  NARYSHKIN NIKOLAI ,  TURPOFF ANTHONY A ,  WEETALL MARLA L ,  WELCH ELLEN ,  WOLL MATTHEW G ,  YANG TIANLE ,  ZHANG NANJING ,  ZHANG XIAOYAN ,  ZHAO XIN ,  GREEN LUKE ,  PINARD EMMANUEL ,  RATNI HASANE

IPC: C07D239/70 ,  A01N43/90 ,  A61K31/519 ,  A61K31/53

Abstract: Provided herein are compounds, compositions thereof and uses therewith for treating spinal muscular atrophy. F////, x-onIntron 6 Exn Intron 7 Eo U Pre-mRNA Spliced mRNA Luciferase +Compound A Activity Eon xon 6 on 7 Exon 8 luc + Yes -Compound Stop Exon 6 Exon 8 luc + No Figure 1

公开(公告)号：AU2012301953C1

公开(公告)日：2016-11-03

申请号：AU2012301953

申请日：2012-08-29

Applicant: PTC THERAPEUTICS INC

Inventor： BRANSTROM ARTHUR ,  JOSYULA VARA PRASAD VENKATA NAGENDRA ,  ARNOLD MICHAEL ANDREW ,  GERASYUTO ALEKSEY I ,  KARP GARY ,  WANG JIASHI ,  BAIRD JOHN DAVID ,  CHEN GUANGMING ,  GINZBURG OLYA ,  NARASIMHAN JANA ,  PEDDI SRINIVASA ,  QI HONGYAN ,  SMITH SEAN WESLEY ,  WOLL MATTHEW G ,  YANG TIANLE ,  ZHANG NANJING ,  ZHANG XIAOYAN

IPC: A01N43/40

Abstract: The present description relates to compounds and forms and pharmaceutical compositions thereof and methods for use thereof to treat or ameliorate bacterial infections caused by wild-type and multi-drug resistant Gram-negative and Gram-positive pathogens.

公开(公告)号：NZ628186A

公开(公告)日：2016-03-31

申请号：NZ62818613

申请日：2013-02-08

Applicant: PTC THERAPEUTICS INC ,  HOFFMANN LA ROCHE

Inventor： GREEN LUKE ,  PINARD EMMANUEL ,  NARASIMHAN JANA ,  WOLL MATTHEW G ,  ZHANG XIAOYAN ,  QI HONGYAN ,  YANG TIANLE ,  CHOI SOONGYU ,  DAKKA AMAL ,  KARP GARY MITCHELL ,  NARYSHKIN NIKOLAI ,  TURPOFF ANTHONY A ,  WEETALL MARLA L ,  WELCH ELLEN ,  ZHANG NANJING ,  ZHAO XIN ,  RATNI HASANE

IPC: A61K31/53 ,  A01N43/90 ,  A61K31/519 ,  C07D239/70

Abstract: Disclosed herein are fused substituted six-membered ring compounds, compositions thereof and uses therewith for treating spinal muscular atrophy. Exemplary compounds include: 2-(3,5-difluoro-4-hydroxyphenyl)-7-(piperazin-1-yl)-4H-pyrido[1,2-a]pyrimidin-4-one hydrochloride; 7-[4-( dimethyl amino)piperidin-1-yl]-2-(3-fluoro-4-methoxyphenyl)-4H-quinolizin-4-one acetate; 2-(2-methyl-1,3-benzothiazol-6-yl)-7-(piperidin-4-yl)-4H-pyrimido[1,2-b]pyridazin-4-one trifluoroacetate (1:1); and 2-(1,3-dimethylpyrrolo[1,2-a]pyrazin-7-yl)-7-(1,2,3,6-tetrahydropyridin-4-yl)-4Hpyrazino[1,2-a ]pyrimidin-4-one hydrochloride (1:2).

公开(公告)号：AU2013216870A1

公开(公告)日：2014-08-28

申请号：AU2013216870

申请日：2013-02-08

Applicant: PTC THERAPEUTICS INC ,  HOFFMANN LA ROCHE

Inventor： QI HONGYAN ,  CHOI SOONGYU ,  DAKKA AMAL ,  KARP GARY MITCHELL ,  NARASIMHAN JANA ,  NARYSHKIN NIKOLAI ,  TURPOFF ANTHONY A ,  WEETALL MARLA L ,  WELCH ELLEN ,  WOLL MATTHEW G ,  YANG TIANLE ,  ZHANG NANJING ,  ZHANG XIAOYAN ,  ZHAO XIN ,  GREEN LUKE ,  PINARD EMMANUEL ,  RATNI HASANE

IPC: C07D239/70 ,  A01N43/90 ,  A61K31/519 ,  A61K31/53

Abstract: Provided herein are compounds, compositions thereof and uses therewith for treating spinal muscular atrophy.

公开(公告)号：CA2863874A1

公开(公告)日：2013-08-15

申请号：CA2863874

申请日：2013-02-08

Applicant: PTC THERAPEUTICS INC ,  HOFFMANN LA ROCHE

Inventor： QI HONGYAN ,  CHOI SOONGYU ,  DAKKA AMAL ,  KARP GARY MITCHELL ,  NARASIMHAN JANA ,  NARYSHKIN NIKOLAI ,  TURPOFF ANTHONY A ,  WEETALL MARIA L ,  WELCH ELLEN ,  WOLL MATTHEW G ,  YANG TIANLE ,  ZHANG NANJING ,  ZHANG XIAOYAN ,  ZHAO XIN ,  GREEN LUKE ,  PINARD EMMANUEL ,  RATNI HASANE

IPC: A61K31/53 ,  A61K31/519 ,  A61K31/551 ,  A61P21/00 ,  A61P25/28 ,  C07D239/70

Abstract: Provided herein are compounds, compositions thereof and uses therewith for treating spinal muscular atrophy.

公开(公告)号：CA2846663A1

公开(公告)日：2013-03-07

申请号：CA2846663

申请日：2012-08-29

Applicant: PTC THERAPEUTICS INC

Inventor： BRANSTROM ARTHUR ,  JOSYULA VARA PRASAD VENKATA NAGENDRA ,  ARNOLD MICHAEL ANDREW ,  GERASYUTO ALEKSEY I ,  KARP GARY ,  WANG JIASHI ,  QI HONGYAN ,  PEDDI SRINIVASA ,  WOLL MATTHEW G ,  SMITH SEAN WESLEY ,  CHEN GUANGMING ,  YANG TIANLE ,  ZHANG NANJING ,  GINZBURG OLYA ,  NARASIMHAN JANA ,  ZHANG XIAOYAN ,  BAIRD JOHN DAVID

IPC: A01N43/40

Abstract: The present description relates to compounds and forms and pharmaceutical compositions thereof and methods for use thereof to treat or ameliorate bacterial infections caused by wild-type and multi-drug resistant Gram-negative and Gram-positive pathogens.

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：UA129258C2

公开(公告)日：2025-03-05

申请号：UAA202008291

申请日：2019-06-25

Applicant: PTC THERAPEUTICS INC

Inventor： SYDORENKO NADIYA ,  ALAM MD RAUFUL ,  ARNOLD MICHAEL A ,  BABU SURESH ,  BHATTACHARYYA ANURADHA ,  CHEN GUANGMING ,  GERASYUTO ALEKSEY I ,  KARP GARY MITCHELL ,  KASSICK ANDREW J ,  MAZZOTTI ANTHONY R ,  MOON YOUNG-CHOON ,  NARASIMHAN JANA ,  PATEL JIGAR ,  TURPOFF ANTHONY ,  WOLL MATTHEW G ,  YAN WUMING ,  ZHANG NANJING

Abstract: Данийвинахідстосуєтьсясполук, їхніхформі фармацевтичнихкомпозиційі способівзастосуваннятакихсполук, їхніхформабокомпозиційдлялікуванняабополегшенняхворобиГентінгтона. . Зокрема, данийвинахідстосуєтьсязаміщенихбіциклічнихгетероциклічнихі гетероарильнихсполукформули (I), їхніхформі фармацевтичнихкомпозиційі способівзастосуваннятакихсполук, їхніхформабокомпозиційдлялікуванняабополегшенняхворобиГентінгтона.

公开(公告)号：DK3814357T3

公开(公告)日：2024-07-22

申请号：DK19736566

申请日：2019-06-25

Applicant: PTC THERAPEUTICS INC

Inventor： SYDORENKO NADIYA ,  ALAM MD RAUFUL ,  ARNOLD MICHAEL A ,  BABU SURESH ,  BHATTACHARYYA ANURADHA ,  CHEN GUANGMING ,  GERASYUTO ALEKSEY I ,  KARP GARY MITCHELL ,  KASSICK ANDREW J ,  MAZZOTTI ANTHONY R ,  MOON YOUNG-CHOON ,  NARASIMHAN JANA ,  PATEL JIGAR ,  TURPOFF ANTHONY ,  WOLL MATTHEW G ,  YAN WUMING ,  ZHANG NANJING

IPC: C07D487/04 ,  A61K31/4985 ,  A61K31/5025 ,  A61P25/00 ,  A61P25/14 ,  A61P25/28 ,  C07D491/04 ,  C07D495/04 ,  C07D519/00

公开(公告)号：AU2023203241A1

公开(公告)日：2023-06-15

申请号：AU2023203241

申请日：2023-05-23

Applicant: PTC THERAPEUTICS INC

Inventor： SYDORENKO NADIYA ,  ALAM MD RAUFUL ,  ARNOLD MICHAEL A ,  BABU SURESH ,  BHATTACHARYYA ANURADHA ,  CHEN GUANGMING ,  GERASYUTO ALEKSEY I ,  KARP GARY MITCHELL ,  KASSICK ANDREW J ,  MAZZOTTI ANTHONY R ,  MOON YOUNG-CHOON ,  NARASIMHAN JANA ,  PATEL JIGAR ,  TURPOFF ANTHONY ,  WOLL MATTHEW G ,  YAN WUMING ,  ZHANG NANJING

IPC: C07D487/04 ,  A61K31/4985 ,  A61K31/5025 ,  A61P25/00 ,  A61P25/14 ,  A61P25/28 ,  C07D491/04 ,  C07D495/04 ,  C07D519/00

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. Formula (I). In particular, the present description relates to substituted bicyclic heterocyclic and heteroaryl compounds 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.