ES2983003T3 - Compuestos heterocíclicos y heteroarílicos para el tratamiento de la enfermedad de Huntington - Google Patents

Compuestos heterocíclicos y heteroarílicos para el tratamiento de la enfermedad de Huntington Download PDF

Info

Publication number
ES2983003T3
ES2983003T3 ES19736566T ES19736566T ES2983003T3 ES 2983003 T3 ES2983003 T3 ES 2983003T3 ES 19736566 T ES19736566 T ES 19736566T ES 19736566 T ES19736566 T ES 19736566T ES 2983003 T3 ES2983003 T3 ES 2983003T3
Authority
ES
Spain
Prior art keywords
phenol
tetramet
daz
azolo
pyr
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Active
Application number
ES19736566T
Other languages
English (en)
Spanish (es)
Inventor
Nadiya Sydorenko
Md Rauful Alam
Michael A Arnold
Suresh Babu
Anuradha Bhattacharyya
Guangming Chen
Aleksey I Gerasyuto
Gary Mitchell Karp
Andrew J Kassick
Anthony R Mazzotti
Young-Choon Moon
Jana Narasimhan
Jigar Patel
Anthony Turpoff
Matthew G Woll
Wuming Yan
Nanjing Zhang
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
PTC Therapeutics Inc
Original Assignee
PTC Therapeutics Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by PTC Therapeutics Inc filed Critical PTC Therapeutics Inc
Application granted granted Critical
Publication of ES2983003T3 publication Critical patent/ES2983003T3/es
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D487/00—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups C07D451/00 - C07D477/00
    • C07D487/02—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups C07D451/00 - C07D477/00 in which the condensed system contains two hetero rings
    • C07D487/04—Ortho-condensed systems
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/4985—Pyrazines or piperazines ortho- or peri-condensed with heterocyclic ring systems
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/50—Pyridazines; Hydrogenated pyridazines
    • A61K31/5025—Pyridazines; Hydrogenated pyridazines ortho- or peri-condensed with heterocyclic ring systems
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D491/00—Heterocyclic compounds containing in the condensed ring system both one or more rings having oxygen atoms as the only ring hetero atoms and one or more rings having nitrogen atoms as the only ring hetero atoms, not provided for by groups C07D451/00 - C07D459/00, C07D463/00, C07D477/00 or C07D489/00
    • C07D491/02—Heterocyclic compounds containing in the condensed ring system both one or more rings having oxygen atoms as the only ring hetero atoms and one or more rings having nitrogen atoms as the only ring hetero atoms, not provided for by groups C07D451/00 - C07D459/00, C07D463/00, C07D477/00 or C07D489/00 in which the condensed system contains two hetero rings
    • C07D491/04—Ortho-condensed systems
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D491/00—Heterocyclic compounds containing in the condensed ring system both one or more rings having oxygen atoms as the only ring hetero atoms and one or more rings having nitrogen atoms as the only ring hetero atoms, not provided for by groups C07D451/00 - C07D459/00, C07D463/00, C07D477/00 or C07D489/00
    • C07D491/02—Heterocyclic compounds containing in the condensed ring system both one or more rings having oxygen atoms as the only ring hetero atoms and one or more rings having nitrogen atoms as the only ring hetero atoms, not provided for by groups C07D451/00 - C07D459/00, C07D463/00, C07D477/00 or C07D489/00 in which the condensed system contains two hetero rings
    • C07D491/04—Ortho-condensed systems
    • C07D491/044—Ortho-condensed systems with only one oxygen atom as ring hetero atom in the oxygen-containing ring
    • C07D491/048—Ortho-condensed systems with only one oxygen atom as ring hetero atom in the oxygen-containing ring the oxygen-containing ring being five-membered
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D491/00—Heterocyclic compounds containing in the condensed ring system both one or more rings having oxygen atoms as the only ring hetero atoms and one or more rings having nitrogen atoms as the only ring hetero atoms, not provided for by groups C07D451/00 - C07D459/00, C07D463/00, C07D477/00 or C07D489/00
    • C07D491/02—Heterocyclic compounds containing in the condensed ring system both one or more rings having oxygen atoms as the only ring hetero atoms and one or more rings having nitrogen atoms as the only ring hetero atoms, not provided for by groups C07D451/00 - C07D459/00, C07D463/00, C07D477/00 or C07D489/00 in which the condensed system contains two hetero rings
    • C07D491/08—Bridged systems
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D495/00—Heterocyclic compounds containing in the condensed system at least one hetero ring having sulfur atoms as the only ring hetero atoms
    • C07D495/02—Heterocyclic compounds containing in the condensed system at least one hetero ring having sulfur atoms as the only ring hetero atoms in which the condensed system contains two hetero rings
    • C07D495/04—Ortho-condensed systems
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D519/00—Heterocyclic compounds containing more than one system of two or more relevant hetero rings condensed among themselves or condensed with a common carbocyclic ring system not provided for in groups C07D453/00 or C07D455/00

Landscapes

  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Health & Medical Sciences (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • General Health & Medical Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Psychology (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Epidemiology (AREA)
  • Nitrogen Condensed Heterocyclic Rings (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Nitrogen And Oxygen Or Sulfur-Condensed Heterocyclic Ring Systems (AREA)
  • Plural Heterocyclic Compounds (AREA)
  • Heterocyclic Carbon Compounds Containing A Hetero Ring Having Oxygen Or Sulfur (AREA)
ES19736566T 2018-06-27 2019-06-25 Compuestos heterocíclicos y heteroarílicos para el tratamiento de la enfermedad de Huntington Active ES2983003T3 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862690653P 2018-06-27 2018-06-27
PCT/US2019/038889 WO2020005873A1 (en) 2018-06-27 2019-06-25 Heterocyclic and heteroaryl compounds for treating huntington's disease

Publications (1)

Publication Number Publication Date
ES2983003T3 true ES2983003T3 (es) 2024-10-21

Family

ID=67180896

Family Applications (1)

Application Number Title Priority Date Filing Date
ES19736566T Active ES2983003T3 (es) 2018-06-27 2019-06-25 Compuestos heterocíclicos y heteroarílicos para el tratamiento de la enfermedad de Huntington

Country Status (30)

Country Link
US (1) US11858941B2 (OSRAM)
EP (2) EP3814357B1 (OSRAM)
JP (1) JP7421507B2 (OSRAM)
KR (1) KR102823158B1 (OSRAM)
CN (1) CN112654625B (OSRAM)
AR (1) AR119651A1 (OSRAM)
AU (2) AU2019294478B2 (OSRAM)
BR (1) BR112020026545A2 (OSRAM)
CA (1) CA3103976A1 (OSRAM)
CL (3) CL2020003378A1 (OSRAM)
CO (1) CO2020016285A2 (OSRAM)
DK (1) DK3814357T3 (OSRAM)
EA (1) EA202092896A1 (OSRAM)
ES (1) ES2983003T3 (OSRAM)
FI (1) FI3814357T3 (OSRAM)
HR (1) HRP20240935T1 (OSRAM)
HU (1) HUE068060T2 (OSRAM)
IL (1) IL279688B2 (OSRAM)
LT (1) LT3814357T (OSRAM)
MX (1) MX2020014098A (OSRAM)
PE (1) PE20211378A1 (OSRAM)
PL (1) PL3814357T3 (OSRAM)
PT (1) PT3814357T (OSRAM)
RS (1) RS65727B1 (OSRAM)
SG (1) SG11202012674PA (OSRAM)
SI (1) SI3814357T1 (OSRAM)
SM (1) SMT202400286T1 (OSRAM)
UA (1) UA129258C2 (OSRAM)
WO (1) WO2020005873A1 (OSRAM)
ZA (1) ZA202100208B (OSRAM)

Families Citing this family (40)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX391850B (es) 2015-12-10 2025-03-19 Ptc Therapeutics Inc Compuestos para usarse en el tratamiento o mejoramiento de la enfermedad de huntington
SG11201911615WA (en) 2017-06-05 2020-01-30 Ptc Therapeutics Inc Compounds for treating huntington's disease
MX2019015580A (es) 2017-06-28 2020-07-28 Ptc Therapeutics Inc Metodos para tratar la enfermedad de huntington.
WO2019005980A1 (en) 2017-06-28 2019-01-03 Ptc Therapeutics, Inc. METHODS OF TREATING HUNTINGTON'S DISEASE
CN111499615B (zh) 2017-08-04 2024-02-02 斯基霍克疗法公司 用于调节剪接的方法和组合物
JP7569688B2 (ja) 2017-12-22 2024-10-18 ハイバーセル，インコーポレイテッド ホスファチジルイノシトールリン酸キナーゼ阻害剤としてのアミノピリジン誘導体
AU2019243048A1 (en) 2018-03-27 2020-10-15 Ptc Therapeutics, Inc. Compounds for treating Huntington's disease
BR112020026534A2 (pt) 2018-06-27 2021-03-23 Ptc Therapeutics, Inc. Compostos de heteroarila para o tratamento da doença de huntington
MX2020014098A (es) 2018-06-27 2021-05-27 Ptc Therapeutics Inc Compuestos heterocíclicos y de heteroarilo para tratar la enfermedad de huntington.
EP3814360B8 (en) 2018-06-27 2024-11-06 PTC Therapeutics, Inc. Heteroaryl compounds for treating huntington's disease
EP3920915A4 (en) 2019-02-05 2022-10-05 Skyhawk Therapeutics, Inc. METHODS AND COMPOSITIONS FOR MODULATING SPLICE
JP7603595B2 (ja) 2019-02-06 2024-12-20 スカイホーク・セラピューティクス・インコーポレーテッド スプライシングを調節するための方法および組成物
KR20210151823A (ko) * 2019-03-15 2021-12-14 스카이호크 테라퓨틱스, 인코포레이티드 비정상적인 스플라이싱을 보정하기 위한 조성물 및 방법
JP7649257B2 (ja) * 2019-05-13 2025-03-19 ピーティーシー セラピューティクス， インコーポレイテッド ハンチントン病を処置するための化合物
TW202112767A (zh) 2019-06-17 2021-04-01 美商佩特拉製藥公司 作為磷脂酸肌醇磷酸激酶抑制劑之胺基吡啶衍生物
US11129829B2 (en) 2019-06-17 2021-09-28 Skyhawk Therapeutics, Inc. Methods for modulating splicing
TW202131920A (zh) 2019-11-01 2021-09-01 瑞士商諾華公司 剪接調節子用於減慢杭丁頓氏舞蹈症進展的治療之用途
JP7736700B2 (ja) * 2020-02-28 2025-09-09 リミックス セラピューティクス インコーポレイテッド 複素環アミド及びスプライシングを調節するためのその使用
IL297044A (en) * 2020-04-09 2022-12-01 Ptc Therapeutics Inc Compounds for the treatment of Huntington's disease
US11806346B2 (en) 2020-05-13 2023-11-07 Chdi Foundation, Inc. HTT modulators for treating Huntington's disease
TW202216725A (zh) 2020-07-02 2022-05-01 美商雷密克斯醫療公司 調控剪接之化合物及方法
CA3183321A1 (en) 2020-07-02 2022-02-06 Remix Therapeutics Inc. 2-(indazol-5-yl)-6-(piperidin-4-yl)-1,7-naphthyridine derivatives and related compounds as modulators for splicing nucleic acids and for the treatment of proliferative diseases
WO2022060944A1 (en) * 2020-09-16 2022-03-24 Skyhawk Therapeutics, Inc. Compositions for modulating splicing
WO2022060943A1 (en) * 2020-09-16 2022-03-24 Skyhawk Therapeutics, Inc. Compositions for modulating splicing
WO2022060951A1 (en) * 2020-09-16 2022-03-24 Skyhawk Therapeutics, Inc. Compositions for modulating splicing
MX2023005541A (es) * 2020-11-12 2023-06-15 Ptc Therapeutics Inc Nuevo transcripto de arn.
JP2023549577A (ja) * 2020-11-13 2023-11-27 ピーティーシー セラピューティクス， インコーポレイテッド ハンチントン病の処置において使用するための錠剤及びそれを作製する方法
EP4308572B1 (en) * 2021-03-17 2025-04-23 F. Hoffmann-La Roche AG New thienopyrimidinone derivatives
TW202304446A (zh) 2021-03-29 2023-02-01 瑞士商諾華公司 剪接調節子用於減慢杭丁頓氏舞蹈症進展的治療之用途
WO2023009816A1 (en) 2021-07-30 2023-02-02 Ptc Therapeutics, Inc. Heteroaryl compounds for treating huntington's disease
WO2023081860A1 (en) * 2021-11-04 2023-05-11 Skyhawk Therapeutics, Inc. Triazine amino derivatives for treating sca3
AU2022394333A1 (en) 2021-11-17 2024-06-27 Chdi Foundation, Inc. HTT modulators for treating Huntington’s disease
CA3252644A1 (en) * 2022-05-10 2023-11-16 Foghorn Therapeutics Inc. PYRAZINE DERIVATIVES AND THEIR USES
KR20250034045A (ko) * 2022-06-15 2025-03-10 피티씨 테라퓨틱스, 인크. 헌팅턴병 치료를 위한 헤테로사이클릭 및 헤테로아릴 화합물
EP4610258A1 (en) 2022-10-26 2025-09-03 Tenvie Therapeutics, Inc. Fused pyridazine derivative
AU2024211840A1 (en) 2023-01-24 2025-07-03 Daiichi Sankyo Company, Limited Substituted benzene compound
WO2024233306A1 (en) * 2023-05-09 2024-11-14 Ptc Therapeutics, Inc. Crystalline forms of 2-[3-(2,2,6,6-tetramethylpiperidin-4-yl)-3h-[1,2,3]triazolo[4,5-c]pyridazin-6-yl]-5-(2h-1,2,3-triazol-2-yl)phenol
AR132817A1 (es) * 2023-06-02 2025-07-30 Merck Sharp & Dohme Llc Heterociclos bicíclicos 5,6 insaturados útiles como inhibidores de la proteína receptora 3 similar a nod
WO2024249389A2 (en) * 2023-06-02 2024-12-05 Merck Sharp & Dohme Llc 5,6 saturated bicyclic heterocyles useful as inhibitors of nod-like receptor protein 3
WO2025040014A1 (zh) * 2023-08-18 2025-02-27 英矽智能科技知识产权有限公司 并环化合物及其制备方法和用途

Family Cites Families (194)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
NL297170A (OSRAM) 1963-04-04 1900-01-01
US3558618A (en) 1968-04-01 1971-01-26 Dow Chemical Co Novel 4h-pyrazino(1,2-a)pyrimidine-4-ones
GB1383409A (en) 1972-09-09 1974-02-12 Pfizer Ltd Derivatives of 2-amino- and 4-amino-quinazoline and pharmaceutical compositions containing them
US4122274A (en) 1977-05-25 1978-10-24 Bristol-Myers Company 3-Tetrazolo-5,6,7,8-substituted-pyrido[1,2-a]pyrimidin-4-ones
JPS56150091A (en) 1980-03-28 1981-11-20 Janssen Pharmaceutica Nv 3-(1-piperidinylalkyl)-4h-pyrido(1,2-a)pyrimidine- 4-one derivative and its manufacture
US4342870A (en) 1980-03-28 1982-08-03 Janssen Pharmaceutica N.V. Novel 3-(1-piperidinylalkyl)-4H-pyrido[1,2-a]pyrimidin-4-one derivatives
FR2567518B1 (fr) 1984-07-11 1987-11-13 Sanofi Sa Nouveaux composes a noyau heterocyclique azote, leur preparation et les medicaments qui en contiennent
US5089633A (en) 1987-04-28 1992-02-18 Georgia Tech Research Corporation Substituted isocoumarins
US5599816A (en) 1990-05-02 1997-02-04 Abbott Laboratories Quinolizinone type compounds
EP0640083A1 (en) 1992-05-13 1995-03-01 E.I. Du Pont De Nemours And Company Substituted pyrido 1,2-a]pyrimidinone derivatives as fungicides
EP0698092B1 (en) 1993-05-11 2007-07-25 The University Of North Carolina At Chapel Hill Antisense oligonucleotides which combat aberrant splicing and methods of using the same
IL122296A0 (en) 1995-06-06 1998-04-05 Abbott Lab Quinolizinone type compounds
US5916916A (en) 1996-10-10 1999-06-29 Eli Lilly And Company 1-aryloxy-2-arylnaphthyl compounds, intermediates, compositions, and methods
US5869500A (en) 1996-12-13 1999-02-09 Hoffmann-La Roche Inc. Pyridone compounds useful in treating Alzheimer's disease
CA2269561C (en) 1998-04-22 2007-06-05 Dainippon Ink And Chemicals, Inc. Naphthalene derivative and liquid crystal composition comprising the same
HK1040076A1 (zh) 1998-09-21 2002-05-24 希雷生物化学有限公司 作为整联蛋白抑制剂的喹嗪酮
US6172216B1 (en) 1998-10-07 2001-01-09 Isis Pharmaceuticals Inc. Antisense modulation of BCL-X expression
US6210892B1 (en) 1998-10-07 2001-04-03 Isis Pharmaceuticals, Inc. Alteration of cellular behavior by antisense modulation of mRNA processing
US6214986B1 (en) 1998-10-07 2001-04-10 Isis Pharmaceuticals, Inc. Antisense modulation of bcl-x expression
ATE312820T1 (de) 1999-10-28 2005-12-15 Trine Pharmaceuticals Inc Pumpeninhibitoren zur freisetzung von medikamenten
CN1891693A (zh) 2000-01-24 2007-01-10 基纳西亚股份有限公司 用于治疗的吗啉代基取代的化合物
EP1416972B1 (en) 2000-11-09 2012-02-08 Cold Spring Harbor Laboratory Chimeric molecules to modulate gene expression
US6774134B2 (en) 2000-12-20 2004-08-10 Bristol-Myers Squibb Company Heterocyclic substituted 2-methyl-benzimidazole antiviral agents
PT1355905E (pt) 2000-12-21 2007-05-31 Vertex Pharma Compostos de pirazole úteis como inibidores de proteínaquinase
GB0100624D0 (en) 2001-01-10 2001-02-21 Vernalis Res Ltd Chemical compounds VII
JPWO2002087589A1 (ja) 2001-04-26 2004-08-12 第一製薬株式会社 薬剤排出ポンプ阻害薬
ATE466581T1 (de) 2001-12-07 2010-05-15 Vertex Pharma Verbindungen auf pyrimidin-basis als gsk-3-hemmer
GB0205281D0 (en) 2002-03-06 2002-04-17 Novartis Ag Organic compounds
ATE548354T1 (de) 2002-07-24 2012-03-15 Ptc Therapeutics Inc Ureido-substituierte benzoesäureverbindungen und ihre verwendung für die nonsense-suppression und behandlung von erkrankungen
AU2003262833A1 (en) 2002-08-23 2004-03-11 The Board Of Trustees Of The Leland Stanford Junior University Fluorescent glycosides and methods for their use
PL375025A1 (en) 2002-09-30 2005-11-14 Neurosearch A/S Novel 1,4-diazabicycloalkane derivatives, their preparation and use
US20070078144A1 (en) 2003-01-29 2007-04-05 Stockwell Brent R Agents for treating neurodegenerative diseases
DE602004025698D1 (de) 2003-06-20 2010-04-08 Novartis Vaccines & Diagnostic PYRIDINOi1,2-A PYRIMIDIN-4-ONVERBINDUNGEN ALS MITTEL GEGEN KREBS
GB0315494D0 (en) 2003-07-02 2003-08-06 Biofocus Plc Compounds which bind to the active site of protein kinase enzymes
EP1651631A1 (en) 2003-08-01 2006-05-03 Genelabs Technologies, Inc. Bicyclic imidazol derivatives against flaviviridae
US7160888B2 (en) 2003-08-22 2007-01-09 Warner Lambert Company Llc [1,8]naphthyridin-2-ones and related compounds for the treatment of schizophrenia
US20050074801A1 (en) 2003-09-09 2005-04-07 Monia Brett P. Chimeric oligomeric compounds comprising alternating regions of northern and southern conformational geometry
US7655657B2 (en) 2003-12-22 2010-02-02 Abbott Laboratories Fused bicycloheterocycle substituted quinuclidine derivatives
US7309699B2 (en) 2003-12-22 2007-12-18 Abbott Laboratories 3-Quinuclidinyl amino-substituted biaryl derivatives
US20050137203A1 (en) 2003-12-22 2005-06-23 Jianguo Ji 3-quinuclidinyl amino-substituted biaryl derivatives
US20050245531A1 (en) 2003-12-22 2005-11-03 Abbott Laboratories Fused bicycloheterocycle substituted quinuclidine derivatives
CA2551178C (en) 2003-12-24 2012-11-06 Biota Scientific Management Pty Ltd Polycyclic agents for the treatment of respiratory syncytial virus infections
SE0400184D0 (sv) 2004-01-30 2004-01-30 Fyrkloevern Scandinavia Ab New therapeutical use
TW200536830A (en) 2004-02-06 2005-11-16 Chugai Pharmaceutical Co Ltd 1-(2H)-isoquinolone derivative
JP2007532570A (ja) 2004-04-08 2007-11-15 ニューロジェン・コーポレーション 置換シンノリン−４−イルアミン類
US20080255162A1 (en) 2004-05-04 2008-10-16 Warner-Lambert Company Llc Pyrrolyl Substituted Pyrido[2,3-D]Pyrimidin-7-Ones and Derivatives Thereof as Therapeutic Agents
CN101146798A (zh) 2005-01-21 2008-03-19 詹森药业有限公司 用作雌激素受体调节剂的新的杂环苯并[c]色烯衍生物
US7879992B2 (en) 2005-01-31 2011-02-01 Isis Pharmaceuticals, Inc. Modification of MyD88 splicing using modified oligonucleotides
GB0501999D0 (en) 2005-02-01 2005-03-09 Sentinel Oncology Ltd Pharmaceutical compounds
JP2006219453A (ja) 2005-02-14 2006-08-24 Tokyo Univ Of Pharmacy & Life Science キノリン環を母核とする金属識別型二波長性蛍光分子
US7563601B1 (en) 2005-06-01 2009-07-21 City Of Hope Artificial riboswitch for controlling pre-mRNA splicing
WO2006138418A2 (en) 2005-06-14 2006-12-28 President And Fellows Of Harvard College Improvement of cognitive performance with sirtuin activators
EP2233470B1 (en) 2005-07-04 2011-12-07 High Point Pharmaceuticals, LLC Histamine H3 receptor antagonists
US7473784B2 (en) 2005-08-01 2009-01-06 Bristol-Myers Squibb Company Benzothiazole and azabenzothiazole compounds useful as kinase inhibitors
US8258109B2 (en) 2005-10-20 2012-09-04 Isis Pharmaceuticals, Inc. Compositions and methods for modulation of LMNA expression
WO2007058894A2 (en) 2005-11-10 2007-05-24 The University Of North Carolina At Chapel Hill Splice switching oligomers for tnf superfamily receptors and their use in treatment of disease
BRPI0617772A2 (pt) 2005-12-06 2011-08-09 Neurosearch As composto, composição farmacêutica, e, uso de um composto
CN101360738A (zh) 2005-12-21 2009-02-04 佩因赛普托药物公司 调节门控离子通道的组合物和方法
PT2161038E (pt) 2006-01-26 2014-03-10 Isis Pharmaceuticals Inc Composições e suas utilizações dirigidas à huntingtina
EP1984499B1 (en) 2006-01-27 2015-05-27 Isis Pharmaceuticals, Inc. Oligomeric compounds and compositions for the use in modulation of micrornas
AR059339A1 (es) 2006-02-09 2008-03-26 Chugai Pharmaceutical Co Ltd Derivados de la cumarina para trastornos proliferativos de celulas, composicion farmaceutica y agente terapeutico que los contiene
US8110681B2 (en) 2006-03-17 2012-02-07 The United States Of America As Represented By The Secretary, Department Of Health And Human Services Compounds for the treatment of spinal muscular atrophy and other uses
CA2645581A1 (en) 2006-03-28 2007-10-04 High Point Pharmaceuticals, Llc Benzothiazoles having histamine h3 receptor activity
WO2007130383A2 (en) 2006-04-28 2007-11-15 Northwestern University Compositions and treatments using pyridazine compounds and secretases
US20080247964A1 (en) 2006-05-08 2008-10-09 Yuelian Xu Substituted azaspiro derivatives
WO2007133756A2 (en) * 2006-05-15 2007-11-22 Neurogen Corporation Crf1 receptor ligands comprising heteroaryl fused bicycles
US7910578B2 (en) 2006-05-23 2011-03-22 Neurosearch A/S 8,10-diaza-bicyclo[4.3.1]decane derivatives and their medical use
CA2658462C (en) 2006-07-20 2011-09-27 Amgen Inc. Substituted pyridone compounds and methods of use
WO2008013997A2 (en) 2006-07-27 2008-01-31 The Trustees Of Columbia University In The City Of New York Fluorescent substrates for monoamine transporters as optical false neurotransmitters
DK2066653T3 (da) 2006-08-03 2012-12-10 Rottapharm Spa 6-1H-Imidazoquinazolin- og -quinolinderivater, nye stærke analgetika og antiinflammatoriske midler
MX2009003892A (es) 2006-10-25 2009-04-23 Neurosearch As Compuestos de oxadiazol y tiadiazol y su uso como moduladores del receptor de acetilcolina nicotinico.
US8314119B2 (en) 2006-11-06 2012-11-20 Abbvie Inc. Azaadamantane derivatives and methods of use
KR20100049698A (ko) 2006-12-22 2010-05-12 아벡사 리미티드 바이사이클 피리미디논 및 그 용도
US20080171792A1 (en) 2006-12-28 2008-07-17 Jobdevairakkam Christopher New Use of highly concentrated formulations of 4-phenylbutyrate for treatment of certain disorders
FR2914188B1 (fr) 2007-03-28 2012-06-22 Trophos Nouvelle composition a base d'oxime de cholest-4-en-3-one
EP2014656A3 (en) 2007-06-11 2011-08-24 High Point Pharmaceuticals, LLC New heteocyclic h3 antagonists
PT2193133E (pt) 2007-09-27 2015-10-22 Fundación Ct Nac De Investigaciones Oncológicas Carlos Iii Imidazolotiadiazóis para utilização como inibidores de proteína quinases
EP2215074B1 (en) 2007-09-27 2014-02-19 The United States of America, as Represented by the Secretary, Department of Health and Human Services Isoindoline compounds for the treatment of spinal muscular atrophy and other uses
US8153813B2 (en) 2007-12-20 2012-04-10 Abbott Laboratories Benzothiazole and benzooxazole derivatives and methods of use
WO2009114874A2 (en) 2008-03-14 2009-09-17 Intellikine, Inc. Benzothiazole kinase inhibitors and methods of use
US20090258907A1 (en) 2008-04-09 2009-10-15 Abbott Laboratories Compounds useful as inhibitors of rock kinases
WO2009131958A2 (en) 2008-04-21 2009-10-29 Institute For Oneworld Health Compounds, compositions and methods comprising triazine derivatives
WO2009151546A2 (en) 2008-05-27 2009-12-17 Ptc Therapeutics, Inc. Methods for treating spinal muscular atrophy
KR20110036583A (ko) 2008-06-20 2011-04-07 로타팜 에스.피.에이. 6-1ｈ-이미다조-퀴나졸린 및 퀴놀린 유도체, 신규 ｍａｏ 억제제 및 이미다졸린 수용체 리간드
EP2138493A1 (en) 2008-06-26 2009-12-30 Sanofi-Aventis Substituted pyrimidone derivatives
MX2011000149A (es) 2008-07-02 2011-02-24 Avexa Ltd Compuestos que tienen propiedades antivirales.
WO2010019236A1 (en) 2008-08-13 2010-02-18 Ptc Therapeutics, Inc Methods for treating spinal muscular atrophy
WO2010024903A1 (en) 2008-08-29 2010-03-04 Yangbo Feng BENZO[d]OXAZOLES AND BENZO[d]THIAZOLES AS KINASE INHIBITORS
AU2009303441A1 (en) 2008-10-16 2010-04-22 Schering Corporation Pyrrolidine, piperidine and piperazine derivatives and methods of use thereof
GB2465405A (en) 2008-11-10 2010-05-19 Univ Basel Triazine, pyrimidine and pyridine analogues and their use in therapy
WO2010071819A1 (en) 2008-12-19 2010-06-24 Schering Corporation Bicyclic heterocyclic derivatives and methods of use thereof
AU2010214101A1 (en) 2009-02-11 2011-09-01 Sunovion Pharmaceuticals Inc. Histamine H3 inverse agonists and antagonists and methods of use thereof
FR2945289A1 (fr) 2009-05-11 2010-11-12 Sanofi Aventis Derives de 2-cycloamino-5-(pyridin-4-yl)imidazo°2,1-b! °1,3,4!thiadiazole, leur preparation et leur application en therapeutique
MX2011014019A (es) 2009-06-19 2012-02-22 Abbott Lab Derivados de diazahomoadamantano y sus metodos de uso.
KR102173836B1 (ko) 2009-09-11 2020-11-05 아이오니스 파마수티컬즈, 인코포레이티드 헌팅틴 발현의 조절
WO2011050245A1 (en) 2009-10-23 2011-04-28 Yangbo Feng Bicyclic heteroaryls as kinase inhibitors
WO2011057204A2 (en) 2009-11-06 2011-05-12 The Johns Hopkins University Lrrk2-mediated neuronal toxicity
EP2501231B1 (en) 2009-11-20 2016-12-21 Merck Sharp & Dohme Corp. Quinolizidinone carboxamide m1 receptor positive allosteric modulators
CN102812023A (zh) 2010-01-13 2012-12-05 韩国巴斯德研究所 抗感染吡啶并(1,2-a)嘧啶类
US20130059902A1 (en) 2010-02-08 2013-03-07 Isis Pharmaceuticals, Inc. Methods and compositions useful in treatment of diseases or conditions related to repeat expansion
WO2011097644A2 (en) 2010-02-08 2011-08-11 Isis Pharmaceuticals, Inc. Selective reduction of allelic variants
EP3321361B1 (en) 2010-02-08 2019-03-27 Ionis Pharmaceuticals, Inc. Selective reduction of allelic variants
US8877707B2 (en) 2010-05-24 2014-11-04 Presidio Pharmaceuticals, Inc. Inhibitors of HCV NS5A
KR101900770B1 (ko) 2010-07-19 2018-09-20 아이오니스 파마수티컬즈, 인코포레이티드 근육긴장성 이영양증-단백질 키나제(dmpk) 발현의 조절 방법
WO2012019106A2 (en) 2010-08-06 2012-02-09 Board Of Regents Of The University Of Nebraska Positive and negative modulators of nmda receptors
WO2012075393A2 (en) 2010-12-02 2012-06-07 President And Fellows Of Harvard College Activators of proteasomal degradation and uses thereof
CN102617548A (zh) 2011-01-31 2012-08-01 北京赛林泰医药技术有限公司 作为gpr受体激动剂的双环杂芳基化合物及其组合物和应用
WO2012104823A2 (en) 2011-02-04 2012-08-09 Novartis Ag Pyridopyrimidinone compounds in the treatment of neurodegenerative diseases
US10017764B2 (en) 2011-02-08 2018-07-10 Ionis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleotides and uses thereof
US8703763B2 (en) 2011-03-02 2014-04-22 Hoffmann-La Roche Inc. Bridged piperidine derivatives
US9447075B2 (en) 2011-08-02 2016-09-20 The Brigham And Women's Hospital, Inc. Pyridazine derivatives as EAAT2 activators
US8871756B2 (en) 2011-08-11 2014-10-28 Hoffmann-La Roche Inc. Compounds for the treatment and prophylaxis of Respiratory Syncytial Virus disease
DK2742135T4 (da) 2011-08-11 2020-07-13 Ionis Pharmaceuticals Inc Bindingsmodificerede gapped oligomeriske forbindelser og anvendelser deraf
EP2560008B1 (en) 2011-08-18 2016-11-30 Korea Institute of Science and Technology Pharmaceutical compositions for preventing or treating degenerative brain disease and method of screening the same
EP2751269B1 (en) 2011-08-29 2016-03-23 Ionis Pharmaceuticals, Inc. Methods and compounds useful in conditions related to repeat expansion
US9662314B2 (en) 2011-10-21 2017-05-30 Tufts Medical Center, Inc. Compounds and methods for the treatment of muscular disease, and related screening methods
GB201119538D0 (en) 2011-11-10 2011-12-21 Viral Ltd Pharmaceutical compounds
KR20140097459A (ko) 2011-11-28 2014-08-06 노파르티스 아게 신규 트리플루오로메틸-옥사디아졸 유도체 및 질환의 치료에서의 그의 용도
WO2013101974A1 (en) 2011-12-30 2013-07-04 Ptc Therapeutics, Inc. Compounds for treating spinal muscular atrophy
JP6193888B2 (ja) 2012-01-26 2017-09-06 ピーティーシー セラピューティクス， インコーポレイテッド 脊髄性筋萎縮症を治療するための化合物
BR112014019750B1 (pt) 2012-02-10 2020-03-03 F. Hoffmann-La Roche Ag. Composto, composição farmacêutica e seus usos
MX352962B (es) 2012-03-01 2017-12-15 Ptc Therapeutics Inc Compuestos para tratar atrofia muscular espinal.
US9914722B2 (en) 2012-03-23 2018-03-13 Ptc Therapeutics, Inc. Compounds for treating spinal muscular atrophy
LT2841428T (lt) * 2012-04-24 2018-12-10 Vertex Pharmaceuticals Incorporated Dnr-pk inhibitoriai
EP2872493B1 (en) 2012-07-13 2018-11-14 Indiana University Research and Technology Corporation 5,6,7-trimethoxy 4-phenyl quinolin-2-one derivatives for treatment of spinal muscular atrophy
US8729263B2 (en) 2012-08-13 2014-05-20 Novartis Ag 1,4-disubstituted pyridazine analogs there of and methods for treating SMN-deficiency-related conditions
EP3459549B1 (en) 2012-10-12 2022-04-06 Ionis Pharmaceuticals, Inc. Selective antisense compounds and uses thereof
EP2906255B1 (en) 2012-10-12 2023-02-22 Ionis Pharmaceuticals, Inc. Antisense compounds and uses thereof
EP2911671B1 (en) * 2012-10-25 2018-06-27 Usher III Initiative Pyrazolopyridazines and methods for treating retinal-degenerative diseases and hearing loss associated with usher syndrome
US9227976B2 (en) * 2012-10-25 2016-01-05 Usher Iii Initiative, Inc. Pyrazolopyridazines and methods for treating retinal-degenerative diseases and hearing loss associated with usher syndrome
JP6255711B2 (ja) 2012-11-01 2018-01-10 株式会社リコー エレクトロクロミック化合物、エレクトロクロミック組成物及び表示素子
US9040712B2 (en) 2013-01-23 2015-05-26 Novartis Ag Thiadiazole analogs thereof and methods for treating SMN-deficiency-related-conditions
EP3778618A1 (en) 2013-02-04 2021-02-17 Ionis Pharmaceuticals, Inc. Selective antisense compounds and uses thereof
CN105026398B (zh) 2013-03-05 2018-05-18 默克专利股份公司 用于治疗疾病诸如癌症的三唑并[4,5-d]嘧啶衍生物
RU2015148926A (ru) 2013-05-14 2017-06-15 Ф. Хоффманн-Ля Рош Аг Аза-оксо-индолы для лечения и профилактики респираторно-синцитиальной вирусной инфекции
WO2014209841A2 (en) 2013-06-25 2014-12-31 F. Hoffmann-La Roche Ag Compounds for treating spinal muscular atrophy
EP3027600B1 (en) 2013-07-31 2022-04-06 Novartis AG 1,4-disubstituted pyridazine derivatives and their use for treating smn-deficiency-related conditions
KR20160045063A (ko) 2013-08-19 2016-04-26 에프. 호프만-라 로슈 아게 스크리닝 방법
WO2015095449A1 (en) 2013-12-19 2015-06-25 Ptc Therapeutics, Inc. Methods for modulating the amount rna transcripts
WO2015095446A1 (en) 2013-12-19 2015-06-25 Ptc Therapeutics, Inc. Methods for modulating the amount of rna transcripts
EP3082820B1 (en) 2013-12-19 2022-07-20 PTC Therapeutics, Inc. Methods for modulating the amount of rna transcripts
EP3087067B1 (en) 2013-12-26 2018-10-24 Takeda Pharmaceutical Company Limited 4-(piperrazin-1-yl)-pyrrolidin-2-one compounds as monoacylglycerol lipase (magl) inhibitors
PT3094728T (pt) 2014-01-16 2022-05-19 Wave Life Sciences Ltd Desenho quiral
JP6473457B2 (ja) 2014-01-17 2019-02-20 ノバルティス アーゲー Ｓｈｐ２の活性を阻害するための１−（トリアジン−３−イル／ピリダジン−３−イル）−ピペリジン／ピペラジン誘導体およびその組成物
AR099134A1 (es) 2014-01-24 2016-06-29 Hoffmann La Roche Procedimiento para la preparación de n-[(3-aminooxetán-3-il)metil]-2-(1,1-dioxo-3,5-dihidro-1,4-benzotiazepín-4-il)-6-metil-quinazolín-4-amina
EP3116858B1 (en) 2014-03-14 2020-11-11 RaQualia Pharma Inc. Azaspiro derivatives as trpm8 antagonists
JP2017512834A (ja) 2014-03-19 2017-05-25 アミディス・ダイアグノスティックス・インコーポレイテッド アミロイド標的剤及びその使用方法
SI3143025T1 (sl) 2014-05-15 2020-01-31 F. Hoffmann-La Roche Ag Spojine za zdravljenje spinalne mišične atrofije
GB201410693D0 (en) 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
CN106573811A (zh) 2014-06-17 2017-04-19 诺维信公司 从废水中去除生物磷
RU2725979C2 (ru) 2014-06-25 2020-07-08 Ф. Хоффманн-Ля Рош Аг ИМИДАЗО[1,2-а]ПИРАЗИН-1-ИЛ-БЕНЗАМИДЫ ДЛЯ ЛЕЧЕНИЯ СПИНАЛЬНОЙ МЫШЕЧНОЙ АТРОФИИ
US20180282347A1 (en) 2014-12-02 2018-10-04 Bayer Cropscience Aktiengesellschaft Bicyclic compounds as pest control agents
LT3237618T (lt) 2014-12-24 2019-07-10 Uniqure Ip B.V. Rnri sukeltas hantingtino geno slopinimas
ES2928714T3 (es) 2015-01-16 2022-11-22 Massachusetts Gen Hospital Compuestos para mejorar el empalme del ARNm
JP6884102B2 (ja) 2015-02-09 2021-06-09 エフ・ホフマン−ラ・ロシュ・アクチェンゲゼルシャフト がんの治療のための化合物
GB201502567D0 (en) 2015-02-16 2015-04-01 Sentinel Oncology Ltd Pharmaceutical compounds
WO2016144351A1 (en) 2015-03-11 2016-09-15 E. I. Du Pont De Nemours And Company Heterocycle-substituted bicyclic azole pesticides
GB201506933D0 (en) 2015-04-23 2015-06-10 Sentinel Oncology Ltd Pharmaceutical compounds
EP3298017B1 (en) 2015-05-20 2019-08-14 H. Hoffnabb-La Roche Ag Compounds for treating spinal muscular atrophy
EP4249472A3 (en) 2015-05-30 2023-12-13 PTC Therapeutics, Inc. Methods for modulating rna splicing
MA43072A (fr) 2015-07-22 2018-05-30 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
US10226448B2 (en) 2015-08-03 2019-03-12 Samumed, Llc 3-(1H-pyrrolo[3,2-C]pyridin-2-yl)-1H-pyrazolo[3,4-B]pyridines and therapeutic uses thereof
WO2017059251A1 (en) 2015-10-02 2017-04-06 Incyte Corporation Heterocyclic compounds useful as pim kinase inhibitors
ES2988532T3 (es) 2015-11-12 2024-11-20 Hoffmann La Roche Composiciones para tratar la atrofia muscular espinal
AR106652A1 (es) 2015-11-12 2018-02-07 Hoffmann La Roche Compuestos para tratar la esclerosis lateral amiotrófica
US10383867B2 (en) 2015-11-28 2019-08-20 Russell Dahl Quinoline derivatives and their use for treating endoplasmic reticulum stress-related diseases and disorders
MX391850B (es) * 2015-12-10 2025-03-19 Ptc Therapeutics Inc Compuestos para usarse en el tratamiento o mejoramiento de la enfermedad de huntington
EP3386978B1 (en) 2015-12-10 2021-01-27 H. Hoffnabb-La Roche Ag Bridged piperidine derivatives
US10526345B2 (en) 2016-04-08 2020-01-07 Mankind Pharma Ltd. Compounds as GPR119 agonists
AR108325A1 (es) 2016-04-27 2018-08-08 Samumed Llc Isoquinolin-3-il carboxamidas y preparación y uso de las mismas
MA45270A (fr) 2016-05-04 2017-11-09 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
JP7044375B2 (ja) 2016-05-31 2022-03-30 ボード オブ リージェンツ，ザ ユニバーシティ オブ テキサス システム Ｐｔｐｎ１１の複素環式阻害剤
US20190264267A1 (en) 2016-07-25 2019-08-29 Wave Life Sciences Ltd. Phasing
TWI848901B (zh) * 2016-10-24 2024-07-21 美商傳達治療有限公司 Shp2磷酸酶抑制劑及其使用方法
EP3606562A4 (en) 2017-04-03 2020-11-11 Acceleron Pharma Inc. COMPOSITIONS AND METHODS FOR THE TREATMENT OF SPINAL AMYOTROPHIA
WO2018218133A1 (en) * 2017-05-26 2018-11-29 Relay Therapeutics, Inc. Pyrazolo[3,4-b]pyrazine derivatives as shp2 phosphatase inhibitors
SG11201911615WA (en) 2017-06-05 2020-01-30 Ptc Therapeutics Inc Compounds for treating huntington's disease
WO2019005980A1 (en) 2017-06-28 2019-01-03 Ptc Therapeutics, Inc. METHODS OF TREATING HUNTINGTON'S DISEASE
MX2019015580A (es) 2017-06-28 2020-07-28 Ptc Therapeutics Inc Metodos para tratar la enfermedad de huntington.
CN111499615B (zh) 2017-08-04 2024-02-02 斯基霍克疗法公司 用于调节剪接的方法和组合物
US20200392161A1 (en) 2018-02-21 2020-12-17 Relay Therapeutics, Inc. Shp2 phosphatase inhibitors and methods of use thereof
AU2019240299B2 (en) * 2018-03-21 2023-06-22 D.E. Shaw Research, Llc SHP2 phosphatase inhibitors and methods of use thereof
WO2019183364A1 (en) * 2018-03-21 2019-09-26 Relay Therapeutics, Inc. Pyrazolo[3,4-b]pyrazine shp2 phosphatase inhibitors and methods of use thereof
AU2019243048A1 (en) 2018-03-27 2020-10-15 Ptc Therapeutics, Inc. Compounds for treating Huntington's disease
BR112020026534A2 (pt) 2018-06-27 2021-03-23 Ptc Therapeutics, Inc. Compostos de heteroarila para o tratamento da doença de huntington
EP3814360B8 (en) 2018-06-27 2024-11-06 PTC Therapeutics, Inc. Heteroaryl compounds for treating huntington's disease
MX2020014098A (es) 2018-06-27 2021-05-27 Ptc Therapeutics Inc Compuestos heterocíclicos y de heteroarilo para tratar la enfermedad de huntington.
KR20210151823A (ko) 2019-03-15 2021-12-14 스카이호크 테라퓨틱스, 인코포레이티드 비정상적인 스플라이싱을 보정하기 위한 조성물 및 방법
JP7649257B2 (ja) 2019-05-13 2025-03-19 ピーティーシー セラピューティクス， インコーポレイテッド ハンチントン病を処置するための化合物
WO2021007378A1 (en) 2019-07-11 2021-01-14 Ptc Therapeutics, Inc. Compounds for use in treating huntington's disease
TW202131920A (zh) 2019-11-01 2021-09-01 瑞士商諾華公司 剪接調節子用於減慢杭丁頓氏舞蹈症進展的治療之用途
IL297044A (en) 2020-04-09 2022-12-01 Ptc Therapeutics Inc Compounds for the treatment of Huntington's disease
MX2023005541A (es) 2020-11-12 2023-06-15 Ptc Therapeutics Inc Nuevo transcripto de arn.
WO2023009816A1 (en) 2021-07-30 2023-02-02 Ptc Therapeutics, Inc. Heteroaryl compounds for treating huntington's disease

Also Published As

Publication number Publication date
IL279688A (en) 2021-03-01
CL2021003022A1 (es) 2022-07-01
PT3814357T (pt) 2024-07-11
BR112020026545A2 (pt) 2021-03-23
AR119651A1 (es) 2022-01-05
LT3814357T (lt) 2024-06-25
SMT202400286T1 (it) 2024-09-16
IL279688B1 (en) 2024-09-01
KR20210042265A (ko) 2021-04-19
JP2021528467A (ja) 2021-10-21
JP7421507B2 (ja) 2024-01-24
WO2020005873A1 (en) 2020-01-02
AU2023203241B2 (en) 2025-01-02
CL2022003434A1 (es) 2023-06-02
SI3814357T1 (sl) 2024-09-30
FI3814357T3 (fi) 2024-07-26
AU2019294478A1 (en) 2021-01-21
ZA202100208B (en) 2025-05-28
CL2020003378A1 (es) 2021-06-18
HUE068060T2 (hu) 2024-12-28
CN112654625A (zh) 2021-04-13
PE20211378A1 (es) 2021-07-27
DK3814357T3 (da) 2024-07-22
CA3103976A1 (en) 2020-01-02
EP3814357A1 (en) 2021-05-05
US11858941B2 (en) 2024-01-02
CN112654625B (zh) 2024-08-06
UA129258C2 (uk) 2025-03-05
EP3814357B1 (en) 2024-05-01
RS65727B1 (sr) 2024-08-30
AU2023203241A1 (en) 2023-06-15
US20210238186A1 (en) 2021-08-05
EP4434990A1 (en) 2024-09-25
CO2020016285A2 (es) 2021-01-18
HRP20240935T1 (hr) 2024-11-22
PL3814357T3 (pl) 2024-09-16
AU2019294478B2 (en) 2023-03-23
MX2020014098A (es) 2021-05-27
EA202092896A1 (ru) 2021-04-13
IL279688B2 (en) 2025-01-01
SG11202012674PA (en) 2021-01-28
KR102823158B1 (ko) 2025-06-20

Similar Documents

Publication Publication Date Title
ES2983003T3 (es) Compuestos heterocíclicos y heteroarílicos para el tratamiento de la enfermedad de Huntington
US11780839B2 (en) Compounds for treating Huntington's disease
EP3969446B1 (en) Compounds for treating huntington's disease
US20210380603A1 (en) Heteroaryl compounds for treating huntington's disease
HK40116823A (en) Heterocyclic and heteroaryl compounds for treating huntington's disease
US20240132509A1 (en) Heterocyclic and heteroaryl compounds for treating huntington's disease
EA045009B1 (ru) Гетероциклические и гетероарильные соединения для лечения болезни гентингтона
HK40043148A (en) Heterocyclic and heteroaryl compounds for treating huntington's disease
HK40043148B (en) Heterocyclic and heteroaryl compounds for treating huntington's disease
EA046152B1 (ru) Соединения для лечения болезни гентингтона
EA041957B1 (ru) Соединения для лечения болезни гентингтона
HK40043147A (en) Heteroaryl compounds for treating huntington's disease