BR112014019750A2 - composto, composlção farmacêutlca, método para aumentar a lnclusão de éxon 7 de smn2 em mrna, método para aumentar a quantidade de proteína smn e método para tratar sma em um indivíduo humano em necessidade - Google Patents

composto, composlção farmacêutlca, método para aumentar a lnclusão de éxon 7 de smn2 em mrna, método para aumentar a quantidade de proteína smn e método para tratar sma em um indivíduo humano em necessidade

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Publication number
BR112014019750A2
BR112014019750A2 BR112014019750A BR112014019750A BR112014019750A2 BR 112014019750 A2 BR112014019750 A2 BR 112014019750A2 BR 112014019750 A BR112014019750 A BR 112014019750A BR 112014019750 A BR112014019750 A BR 112014019750A BR 112014019750 A2 BR112014019750 A2 BR 112014019750A2
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BR
Brazil
Prior art keywords
increasing
mrna
inclusion
compound
pharmaceutical composition
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BR112014019750A
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English (en)
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BR112014019750B1 (pt
BR112014019750A8 (pt
Inventor
Dakka Amal
A Turpoff Anthony
Welch Ellen
Pinard Emmanuel
Mitchell Karp Gary
Ratni Hasane
Qi Hongyan
Narasimhan Jana
Green Luke
L Weetall Marla
G Woll Matthew
Zhang Nanjing
Naryshkin Nikolai
Choi Soongyu
Yang Tianle
Zhang Xiaoyan
Zhao Xin
Original Assignee
Hoffmann La Roche
Ptc Therapeutics Inc
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Publication of BR112014019750A2 publication Critical patent/BR112014019750A2/pt
Publication of BR112014019750A8 publication Critical patent/BR112014019750A8/pt
Publication of BR112014019750B1 publication Critical patent/BR112014019750B1/pt

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    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D471/00Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, at least one ring being a six-membered ring with one nitrogen atom, not provided for by groups C07D451/00 - C07D463/00
    • C07D471/02Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, at least one ring being a six-membered ring with one nitrogen atom, not provided for by groups C07D451/00 - C07D463/00 in which the condensed system contains two hetero rings
    • C07D471/04Ortho-condensed systems
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495Heterocyclic 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
    • A61K31/505Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/519Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D239/00Heterocyclic compounds containing 1,3-diazine or hydrogenated 1,3-diazine rings
    • C07D239/70Heterocyclic compounds containing 1,3-diazine or hydrogenated 1,3-diazine rings condensed with carbocyclic rings or ring systems
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D487/00Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups C07D451/00 - C07D477/00
    • C07D487/02Heterocyclic 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
    • C07D487/04Ortho-condensed systems
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D519/00Heterocyclic 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

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  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Health & Medical Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurology (AREA)
  • Epidemiology (AREA)
  • Biomedical Technology (AREA)
  • Neurosurgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Nitrogen Condensed Heterocyclic Rings (AREA)
  • Nitrogen And Oxygen Or Sulfur-Condensed Heterocyclic Ring Systems (AREA)
  • Heterocyclic Carbon Compounds Containing A Hetero Ring Having Nitrogen And Oxygen As The Only Ring Hetero Atoms (AREA)
  • Steroid Compounds (AREA)
  • Pyridine Compounds (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
BR112014019750-4A 2012-02-10 2013-02-08 Composto, composição farmacêutica e seus usos BR112014019750B1 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201261597523P 2012-02-10 2012-02-10
US61/597,523 2012-02-10
PCT/US2013/025292 WO2013119916A2 (en) 2012-02-10 2013-02-08 Compounds for treating spinal muscular atrophy

Publications (3)

Publication Number Publication Date
BR112014019750A2 true BR112014019750A2 (pt) 2017-06-20
BR112014019750A8 BR112014019750A8 (pt) 2018-01-16
BR112014019750B1 BR112014019750B1 (pt) 2020-03-03

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US (6) US9586955B2 (pt)
EP (2) EP2812004B1 (pt)
JP (2) JP6092897B2 (pt)
KR (2) KR102137087B1 (pt)
CN (2) CN104349777B (pt)
AR (1) AR092794A1 (pt)
AU (2) AU2013216870B2 (pt)
BR (1) BR112014019750B1 (pt)
CA (1) CA2863874C (pt)
CL (1) CL2014002100A1 (pt)
CO (1) CO7061082A2 (pt)
CR (1) CR20140376A (pt)
DK (1) DK2812004T3 (pt)
EA (2) EA037123B1 (pt)
EC (1) ECSP14017269A (pt)
ES (1) ES2697174T3 (pt)
HK (1) HK1202077A1 (pt)
HU (1) HUE039779T2 (pt)
IL (2) IL233959A (pt)
MA (1) MA35920B1 (pt)
MX (2) MX354074B (pt)
NZ (1) NZ628186A (pt)
PE (1) PE20142364A1 (pt)
PH (2) PH12014501786B1 (pt)
PL (1) PL2812004T3 (pt)
SG (2) SG10201609188WA (pt)
TR (1) TR201813877T4 (pt)
TW (2) TWI585085B (pt)
UA (1) UA116981C2 (pt)
WO (1) WO2013119916A2 (pt)

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