公开(公告)号：IL256029D0

公开(公告)日：2018-01-31

申请号：IL25602917

申请日：2017-11-30

Applicant: PTC THERAPEUTICS INC ,  BAIAZITOV RAMIL Y

Inventor： BAIAZITOV RAMIL Y

IPC: C07H20060101

Abstract: Methods of using and pharmaceutical compositions comprising (2R,3S,4R,5R,6S)-5-amino-6-(((1R,2S,3S,4R,6S)-4,6-diamino-3-(((2R,3R,4R,5R)-3,5-dihydroxy-5-methyl-4-(methylamino)tetrahydro-2H-pyran-2- yl)oxy)-2-hydroxycyclohexyl)oxy)-2-(hydroxymethyl)tetrahydro-2H-pyran-3,4-diol are disclosed, including methods of treating or preventing a nonsense mutation mediated disease associated with premature translation termination or a premature stop codon resulting from a germline or somatic nonsense mutation.

公开(公告)号：CA3094700A1

公开(公告)日：2019-10-03

申请号：CA3094700

申请日：2019-03-27

Applicant: PTC THERAPEUTICS INC

Inventor： SYDORENKO NADIYA ,  AMEDZO LUKIANA ,  BABU SURESH ,  BAIAZITOV RAMIL Y ,  BARRAZA SCOTT J ,  BHATTACHARYYA ANURADHA ,  KARP GARY MITCHELL ,  KENTON NATHANIEL T ,  MAZZOTTI ANTHONY R ,  MOON YOUNG-CHOON ,  MSZAR NICHOLAS W ,  NARASIMHAN JANA ,  PAL SUDIPTA ,  PATEL JIGAR S ,  TURPOFF ANTHONY ,  WOLL MATHEW G ,  XU ZHENRONG ,  ZHANG NANJING ,  ALAM RAUFUL

IPC: A61K31/395 ,  A61K31/53 ,  A61K47/10 ,  C07D251/12 ,  C07D251/16 ,  C07D401/02

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) or Formula (II), 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：MX2020009956A

公开(公告)日：2021-01-15

申请号：MX2020009956

申请日：2019-03-27

Applicant: PTC THERAPEUTICS INC

Inventor： WOLL MATTHEW G ,  NARASIMHAN JANA ,  ZHANG NANJING ,  KARP GARY MITCHELL ,  SYDORENKO NADIYA ,  BABU SURESH ,  BHATTACHARYYA ANURADHA ,  BARRAZA SCOTT J ,  MAZZOTTI ANTHONY R ,  TURPOFF ANTHONY ,  XU ZHENRONG ,  MOON YOUNG-CHOON ,  PATEL JIGAR S ,  ALAM MD RAUFUL ,  AMEDZO LUKIANA ,  BAIAZITOV RAMIL Y ,  KENTON NATHANIEL T ,  MSZAR NICHOLAS W ,  PAL SUDIPTA

IPC: A61K31/395 ,  A61K31/53 ,  A61K47/10 ,  C07D251/12 ,  C07D251/16 ,  C07D401/02

Abstract: La descripción actual se refiere a compuestos, formas, y composiciones farmacéuticas de los mismos y métodos para usar tales compuestos, formas, o composiciones de los mismos para el tratamiento o aminoración de la enfermedad de Huntington. (ver Fórmulas) En particular, la descripción actual se refiere a compuestos heteroarilo monocíclicos sustituidos de la Fórmula (I) o Fórmula (II), formas y composiciones farmacéuticas de los mismos y métodos para usar tales compuestos, formas, o composiciones de los mismos para el tratamiento o aminoración de la enfermedad de Hungtinton.

公开(公告)号：AU2019243943A1

公开(公告)日：2020-10-15

申请号：AU2019243943

申请日：2019-03-27

Applicant: PTC THERAPEUTICS INC

Inventor： SYDORENKO NADIYA ,  ALAN MD RAUFUL ,  AMEDZO LUKIANA ,  BABU SURESH ,  BAIAZITOV RAMIL Y ,  BARRAZA SCOTT J ,  BHATTACHARYYA ANURADHA ,  KARP GARY MITCHELL ,  KENTON NATHANIEL T ,  MAZZOTTI ANTHONY R ,  MOON YOUNG-CHOON ,  MSZAR NICHOLAS W ,  NARASIMHAN JANA ,  PAL SUDIPTA ,  PATEL JIGAR S ,  TURPOFF ANTHONY ,  WOLL MATHEW G ,  XU ZHENRONG ,  ZHANG NANJING

IPC: A61K31/395 ,  A61K31/53 ,  A61K47/10 ,  C07D251/12 ,  C07D251/16 ,  C07D401/02

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) or Formula (II), 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：IL256029A

公开(公告)日：2020-09-30

申请号：IL25602917

申请日：2017-11-30

Applicant: PTC THERAPEUTICS INC ,  BAIAZITOV RAMIL Y

Inventor： BAIAZITOV RAMIL Y

IPC: A61K31/7036 ,  A61K45/06 ,  A61P35/00 ,  A61P43/00 ,  C07H15/236

Abstract: Methods of using and pharmaceutical compositions comprising (2R,3S,4R,5R,6S)-5-amino-6-(((1R,2S,3S,4R,6S)-4,6-diamino-3-(((2R,3R,4R,5R)-3,5-dihydroxy-5-methyl-4-(methylamino)tetrahydro-2H-pyran-2- yl)oxy)-2-hydroxycyclohexyl)oxy)-2-(hydroxymethyl)tetrahydro-2H-pyran-3,4-diol are disclosed, including methods of treating or preventing a nonsense mutation mediated disease associated with premature translation termination or a premature stop codon resulting from a germline or somatic nonsense mutation.

公开(公告)号：CA2987980A1

公开(公告)日：2016-12-08

申请号：CA2987980

申请日：2016-06-03

Applicant: PTC THERAPEUTICS INC

Inventor： BAIAZITOV RAMIL Y

IPC: C07H15/23

Abstract: Methods of using and pharmaceutical compositions comprising (2R,3S,4R,5R,6S)-5- amino-6-(((lR,2S,3S,4R,6S)-4,6-diamino-3-(((2R,3R,4R,5R)-3,5-dihydroxy-5-methyl-4-(methylamino)tetrahydro-2H-pyran-2-yl)oxy)-2-hydroxycyclohexyl)oxy)-2- (hydroxymethyl)tetrahydro-2H-pyran-3,4-diol are disclosed, including methods of treating or preventing a nonsense mutation mediated disease associated with premature translation termination or a premature stop codon resulting from a germline or somatic nonsense mutation.