NO20075682L - Sammensetninger omfattende et oralt aktivt 1,2,4-oksadiazol for nonsense mutasjonsundertrykkelsesterapi - Google Patents
Sammensetninger omfattende et oralt aktivt 1,2,4-oksadiazol for nonsense mutasjonsundertrykkelsesterapiInfo
- Publication number
- NO20075682L NO20075682L NO20075682A NO20075682A NO20075682L NO 20075682 L NO20075682 L NO 20075682L NO 20075682 A NO20075682 A NO 20075682A NO 20075682 A NO20075682 A NO 20075682A NO 20075682 L NO20075682 L NO 20075682L
- Authority
- NO
- Norway
- Prior art keywords
- oxadiazole
- nonsense
- mutational
- compositions
- orally active
- Prior art date
Links
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- 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
- A61K31/41—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with two or more ring hetero atoms, at least one of which being nitrogen, e.g. tetrazole
- A61K31/4245—Oxadiazoles
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- A—HUMAN NECESSITIES
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- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0053—Mouth and digestive tract, i.e. intraoral and peroral administration
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- A61K9/14—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
- A61K9/141—Intimate drug-carrier mixtures characterised by the carrier, e.g. ordered mixtures, adsorbates, solid solutions, eutectica, co-dried, co-solubilised, co-kneaded, co-milled, co-ground products, co-precipitates, co-evaporates, co-extrudates, co-melts; Drug nanoparticles with adsorbed surface modifiers
- A61K9/145—Intimate drug-carrier mixtures characterised by the carrier, e.g. ordered mixtures, adsorbates, solid solutions, eutectica, co-dried, co-solubilised, co-kneaded, co-milled, co-ground products, co-precipitates, co-evaporates, co-extrudates, co-melts; Drug nanoparticles with adsorbed surface modifiers with organic compounds
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- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
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- Hospice & Palliative Care (AREA)
- Psychiatry (AREA)
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Abstract
Den foreliggende oppfinnelsen er relatert til spesifikke doser og doseringskurer for å benytte 1,2,4-oxadiazol benzosyreforbindelse i behandling eller forebygging av sykdommer assosiert med nonsens mutasjoner. Nærmere bestemt er oppfinnelsen relatert til spesifikke doser og doseringskurer for anvendelsen av 3-[5-(2-fluorfenyl)[1,2,4]oxadiazol-3-yl]-benzosyre i pattedyr som har sykdommer assosiert med nonsens mutasjoner.
Priority Applications (1)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
NO20170035A NO340617B1 (no) | 2005-04-08 | 2017-01-09 | Farmasøytisk sammensetning omfattende i form av granuler i en enhetsdoseringsformulering en 1,2,4-oxadiazol benzosyreforbindelse for behandling av en sykdom assosiert med et prematurt stoppcodon |
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US66978905P | 2005-04-08 | 2005-04-08 | |
US78739506P | 2006-03-30 | 2006-03-30 | |
PCT/US2006/012887 WO2006110483A1 (en) | 2005-04-08 | 2006-04-06 | Compositions of an orally active 1,2,4-oxadiazole for nonsense mutation suppression therapy |
Publications (2)
Publication Number | Publication Date |
---|---|
NO20075682L true NO20075682L (no) | 2008-01-07 |
NO339909B1 NO339909B1 (no) | 2017-02-13 |
Family
ID=36763092
Family Applications (2)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
NO20075682A NO339909B1 (no) | 2005-04-08 | 2007-11-07 | Sammensetninger av et oralt aktivt 1,2,4-oxadiazol for ”nonsens” mutasjon undertrykkende terapi |
NO20170035A NO340617B1 (no) | 2005-04-08 | 2017-01-09 | Farmasøytisk sammensetning omfattende i form av granuler i en enhetsdoseringsformulering en 1,2,4-oxadiazol benzosyreforbindelse for behandling av en sykdom assosiert med et prematurt stoppcodon |
Family Applications After (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
NO20170035A NO340617B1 (no) | 2005-04-08 | 2017-01-09 | Farmasøytisk sammensetning omfattende i form av granuler i en enhetsdoseringsformulering en 1,2,4-oxadiazol benzosyreforbindelse for behandling av en sykdom assosiert med et prematurt stoppcodon |
Country Status (17)
Country | Link |
---|---|
US (7) | US8716321B2 (no) |
EP (2) | EP2402002B1 (no) |
JP (3) | JP5800451B2 (no) |
CN (1) | CN103720688A (no) |
AU (1) | AU2006235115C1 (no) |
BR (1) | BRPI0609089A2 (no) |
CA (1) | CA2603402C (no) |
DK (2) | DK2402002T3 (no) |
ES (2) | ES2390804T3 (no) |
HK (1) | HK1111897A1 (no) |
IL (2) | IL186433A (no) |
MX (1) | MX2007012206A (no) |
NO (2) | NO339909B1 (no) |
NZ (1) | NZ562197A (no) |
PL (2) | PL1874306T3 (no) |
PT (2) | PT2402002T (no) |
WO (1) | WO2006110483A1 (no) |
Families Citing this family (49)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
ZA200508298B (en) | 2003-04-11 | 2007-01-31 | Ptc Therapeutics Inc | 1,2,4-oxadiazole benzoic acid compounds |
US8716321B2 (en) | 2005-04-08 | 2014-05-06 | Ptc Therapeutics, Inc. | Methods for dosing an orally active 1,2,4-oxadiazole |
MX2008012515A (es) | 2006-03-30 | 2008-11-19 | Ptc Therapeutics Inc | Metodos para la produccion de proteina funcional a partir de adn que tiene una mutacion sin sentido y el tratamiento de trastornos asociados con la misma. |
US7645789B2 (en) | 2006-04-07 | 2010-01-12 | Vertex Pharmaceuticals Incorporated | Indole derivatives as CFTR modulators |
PL3091011T3 (pl) | 2006-04-07 | 2018-06-29 | Vertex Pharmaceuticals Incorporated | Modulatory transporterów posiadających kasetę wiążącą ATP |
US10022352B2 (en) | 2006-04-07 | 2018-07-17 | Vertex Pharmaceuticals Incorporated | Modulators of ATP-binding cassette transporters |
NZ598012A (en) | 2006-09-08 | 2013-08-30 | Ptc Therapeutics Inc | Processes for the preparation of 1,2,4-oxadiazole benzoic acids |
WO2008130370A1 (en) | 2006-09-25 | 2008-10-30 | Ptc Therapeutics, Inc. | Hydroxylated 1,2,4-oxadiazole benzoic acid compounds, compositions thereof and the use for nonsense suppression |
CL2007002743A1 (es) * | 2006-09-25 | 2008-07-11 | Pct Therapeutics Inc Soc Organ | Forma cristalina del acido 3-[5-(2-fluorofenil)-[1,2,4]oxadiazol-3-il]-benzoico; composicion farmaceutica que comprende a dicha forma cristalina; y su uso para modular la terminacion prematura de traslado o decaimiento de marn, tales como carcicoma. |
AU2016262645B2 (en) * | 2006-10-12 | 2018-05-17 | Ptc Therapeutics, Inc. | Methods for dosing an orally active 1,2,4-oxadiazole for nonsense mutation suppression therapy |
SI2073805T1 (sl) | 2006-10-12 | 2015-10-30 | Ptc Therapeutics, Inc. | Postopki za odmerjanje peroralno aktivnega 1,2,4-oksadiazola za zdravljenje supresije nesmiselnih mutacij |
US8563573B2 (en) | 2007-11-02 | 2013-10-22 | Vertex Pharmaceuticals Incorporated | Azaindole derivatives as CFTR modulators |
JP2010535708A (ja) | 2007-08-03 | 2010-11-25 | ビオマリン アイジーエー リミテッド | デュシェンヌ型筋ジストロフィーの治療のための薬物併用 |
GB0715087D0 (en) | 2007-08-03 | 2007-09-12 | Summit Corp Plc | Drug combinations for the treatment of duchenne muscular dystrophy |
PL2217572T3 (pl) | 2007-11-16 | 2014-04-30 | Vertex Pharma | Modulatory izochinolinowe transporterów zawierających kasetę wiążącą ATP |
SG186638A1 (en) | 2007-12-07 | 2013-01-30 | Vertex Pharma | Solid forms of 3-(6-(1-(2,2-difluorobenzo[d][1,3] dioxol-5-yl) cyclopropanecarboxamido)-3-methylpyridin-2-yl) benzoic acid |
ES2442945T3 (es) | 2008-03-31 | 2014-02-14 | Vertex Pharmaceuticals Inc. | Derivados de piridilo como moduladores del CFTR |
US8802868B2 (en) | 2010-03-25 | 2014-08-12 | Vertex Pharmaceuticals Incorporated | Solid forms of (R)-1(2,2-difluorobenzo[D][1,3]dioxo1-5-yl)-N-(1-(2,3-dihydroxypropyl-6-fluoro-2-(1-hydroxy-2-methylpropan2-yl)-1H-Indol-5-yl)-Cyclopropanecarboxamide |
ES2845449T3 (es) | 2010-03-25 | 2021-07-26 | Vertex Pharma | Dispersión sólida de forma amorfa de (R)-1(2,2-difluorobenzo[d][1,3]dioxol-5-il)-N-(1-(2,3-dihidroxipropil)-6-fluoro-2(1-hidroxi-2-metilpropan-2-il)-1h-indol-5il)-ciclopropanocarboxamida |
RU2592368C2 (ru) | 2010-04-07 | 2016-07-20 | Вертекс Фармасьютикалз Инкорпорейтед | ФАРМАЦЕВТИЧЕСКИЕ КОМПОЗИЦИИ, ВКЛЮЧАЮЩИЕ 3-(2, 2-ДИФТОРБЕНЗО[d][1, 3]ДИОКСОЛ-5-ИЛ)ЦИКЛОПРОПАНКАРБОКСАМИДО)-3-МЕТИЛПИРИДИН-2-ИЛ)БЕНЗОЙНУЮ КИСЛОТУ, И ИХ ВВЕДЕНИЕ |
TWI549950B (zh) | 2010-04-07 | 2016-09-21 | 維泰克斯製藥公司 | 3-(6-(1-(2,2-二氟苯并[d][1,3]二氧雜環戊烯-5-基)環丙烷甲醯胺基)-3-甲基吡啶-2-基)苯甲酸之固體形式 |
EP3381899B1 (en) | 2010-04-22 | 2021-01-06 | Vertex Pharmaceuticals Incorporated | Intermediate compound for process of producing cycloalkylcarboxamido-indole compounds |
US8563593B2 (en) | 2010-06-08 | 2013-10-22 | Vertex Pharmaceuticals Incorporated | Formulations of (R)-1-(2,2-difluorobenzo[D] [1,3] dioxol-5-yl)-N-(1-(2,3-dihydroxypropyl)-6-fluoro-2-(1-hydroxy-2-methylpropan-2-yl)-1H-indol-5-yl)cyclopropanecarboxamide |
PL2600865T3 (pl) * | 2010-08-05 | 2019-05-31 | Institut National De La Sante Et De La Rech Medicale | Związek użyteczny do leczenia chorób, w których pośredniczy mutacja nonsensowna oraz kompozycje farmaceutyczne zawierające wymieniony związek |
CA2808501A1 (en) | 2010-08-23 | 2012-03-01 | Vertex Pharmaceuticals Incorporated | Pharmaceutical composition of (r)-1-(2,2-difluorobenzo[d][1,3]dioxol-5-yl)-n-(1-(2,3-dihydroxy propyl)-6-fluoro-2-(1-hydroxy-2-methylpropan-2-yl)-1h-indol-5-yl) cyclopropanecarboxamide and administration therof |
SI2776427T1 (sl) | 2011-11-08 | 2017-05-31 | Vertex Pharmaceuticals Incorporated | Modulatorji prenašalcev ATP-vezavne kasete |
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