CL2012000495A1 - Producto farmacéutico para la citoreducción de sustrato de esfingomielina acumulada y tratar deficiencia de esfingo mielinasa acida (asmd). - Google Patents
Producto farmacéutico para la citoreducción de sustrato de esfingomielina acumulada y tratar deficiencia de esfingo mielinasa acida (asmd).Info
- Publication number
- CL2012000495A1 CL2012000495A1 CL2012000495A CL2012000495A CL2012000495A1 CL 2012000495 A1 CL2012000495 A1 CL 2012000495A1 CL 2012000495 A CL2012000495 A CL 2012000495A CL 2012000495 A CL2012000495 A CL 2012000495A CL 2012000495 A1 CL2012000495 A1 CL 2012000495A1
- Authority
- CL
- Chile
- Prior art keywords
- myelinase
- asmd
- deficiency
- pharmaceutical product
- sphingin
- Prior art date
Links
- 239000002253 acid Substances 0.000 title abstract 2
- 230000007812 deficiency Effects 0.000 title abstract 2
- 239000000825 pharmaceutical preparation Substances 0.000 title abstract 2
- 229940127557 pharmaceutical product Drugs 0.000 title abstract 2
- 239000000758 substrate Substances 0.000 title abstract 2
- 230000002435 cytoreductive effect Effects 0.000 title 1
- 208000034012 Acid sphingomyelinase deficiency Diseases 0.000 abstract 1
- 101000595674 Homo sapiens Pituitary homeobox 3 Proteins 0.000 abstract 1
- 102100036088 Pituitary homeobox 3 Human genes 0.000 abstract 1
- 208000026753 anterior segment dysgenesis Diseases 0.000 abstract 1
Classifications
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
- A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
- A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P11/00—Drugs for disorders of the respiratory 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
-
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
- A61P3/06—Antihyperlipidemics
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y301/00—Hydrolases acting on ester bonds (3.1)
- C12Y301/04—Phosphoric diester hydrolases (3.1.4)
- C12Y301/04012—Sphingomyelin phosphodiesterase (3.1.4.12)
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Biomedical Technology (AREA)
- Wood Science & Technology (AREA)
- Genetics & Genomics (AREA)
- Zoology (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Gastroenterology & Hepatology (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Epidemiology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Immunology (AREA)
- Hematology (AREA)
- Obesity (AREA)
- Diabetes (AREA)
- Psychiatry (AREA)
- Hospice & Palliative Care (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Microbiology (AREA)
- Pulmonology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Enzymes And Modification Thereof (AREA)
- Dermatology (AREA)
Abstract
LA INVENCIÓN SE RELACIONA A UN PRODUCTO FARMACÉUTICO PARA LA CITOREDUCCIÓN DE SUSTRATO DE ESFINGOMIELINA ACUMULADA Y TRATAR EN PACIENTES HUMANOS LA DEFICIENCIA DE ESFINGO MIELINASA ACIDA (ASMD), Y EN PARTICULAR, PACIENTES CON MANIFESTACIONES NO-NEUROLÓGICAS DE LA ENFERMEDAD DE NIEMANN-PICK (NPD).
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US23811309P | 2009-08-28 | 2009-08-28 | |
| PCT/US2010/047057 WO2011025996A2 (en) | 2009-08-28 | 2010-08-28 | Dose escalation enzyme replacement therapy for treating acid sphingomyelinase deficiency |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CL2012000495A1 true CL2012000495A1 (es) | 2014-02-07 |
Family
ID=43625265
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CL2012000495A CL2012000495A1 (es) | 2009-08-28 | 2010-08-28 | Producto farmacéutico para la citoreducción de sustrato de esfingomielina acumulada y tratar deficiencia de esfingo mielinasa acida (asmd). |
Country Status (29)
| Country | Link |
|---|---|
| US (9) | US8349319B2 (es) |
| EP (3) | EP3482767B1 (es) |
| JP (8) | JP5955219B2 (es) |
| KR (5) | KR102094253B1 (es) |
| CN (3) | CN105999238A (es) |
| AU (6) | AU2010286550B2 (es) |
| BR (1) | BR112012004377A2 (es) |
| CA (1) | CA2773133A1 (es) |
| CL (1) | CL2012000495A1 (es) |
| CY (1) | CY1122128T1 (es) |
| DK (3) | DK2470199T3 (es) |
| ES (2) | ES2738859T3 (es) |
| FI (2) | FI3998078T3 (es) |
| FR (1) | FR22C1062I2 (es) |
| HR (2) | HRP20191385T1 (es) |
| HU (3) | HUE046598T2 (es) |
| IL (4) | IL314807A (es) |
| LT (3) | LT3482767T (es) |
| LU (1) | LUC00288I2 (es) |
| MX (2) | MX392330B (es) |
| NL (1) | NL301211I2 (es) |
| NO (1) | NO2022054I1 (es) |
| PL (2) | PL3482767T3 (es) |
| PT (3) | PT3998078T (es) |
| RU (2) | RU2731616C2 (es) |
| SI (2) | SI2470199T1 (es) |
| TR (1) | TR201911293T4 (es) |
| WO (1) | WO2011025996A2 (es) |
| ZA (2) | ZA201201435B (es) |
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| Publication number | Priority date | Publication date | Assignee | Title |
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| WO2006007560A2 (en) | 2004-07-01 | 2006-01-19 | University Of Pennsylvania | Targeted protein replacement for the treatment of lysosomal storage disorders |
| US20080228056A1 (en) | 2007-03-13 | 2008-09-18 | Michael Blomquist | Basal rate testing using frequent blood glucose input |
| US7751907B2 (en) | 2007-05-24 | 2010-07-06 | Smiths Medical Asd, Inc. | Expert system for insulin pump therapy |
| US8221345B2 (en) | 2007-05-30 | 2012-07-17 | Smiths Medical Asd, Inc. | Insulin pump based expert system |
| PT3252161T (pt) | 2007-06-06 | 2022-02-01 | Genzyme Corp | Terapia genética para doenças do armazenamento lisossomal |
| KR102094253B1 (ko) * | 2009-08-28 | 2020-03-31 | 이칸 스쿨 오브 메디슨 엣 마운트 시나이 | 산성 스핑고미엘리나제 결핍을 치료하기 위한 투여량 단계적 증가 효소 대체 요법 |
| US8882701B2 (en) | 2009-12-04 | 2014-11-11 | Smiths Medical Asd, Inc. | Advanced step therapy delivery for an ambulatory infusion pump and system |
| WO2011146143A2 (en) | 2010-05-21 | 2011-11-24 | Ohmx Corporation | Detection of cancer by assaying psa enzymatic activity |
| LT2685986T (lt) | 2011-03-18 | 2020-03-10 | Genzyme Corporation | Gliukozilceramido sintazės slopiklis |
| SI2753346T1 (sl) | 2011-09-07 | 2020-09-30 | Mount Sinai School Of Medicine | Ceramidaza in diferenciacija celic |
| JP6243846B2 (ja) | 2011-11-15 | 2017-12-06 | セントジーン アーゲー | ニーマン・ピック病の診断のための方法 |
| HUE051020T2 (hu) | 2012-06-01 | 2021-01-28 | Icahn School Med Mount Sinai | A ceramid szintjei a fertõzések kezelésében és megelõzésében |
| JOP20130273B1 (ar) | 2012-09-11 | 2021-08-17 | Genzyme Corp | مثبطات انزيم (سينثاز) غلوكوسيل سيراميد |
| WO2014160390A1 (en) | 2013-03-14 | 2014-10-02 | Icahn School Of Medicine At Mount Sinai | Therapeutic acid ceramidase compositions and methods of making and using them |
| JO3713B1 (ar) | 2013-03-15 | 2021-01-31 | Genzyme Corp | أشكال ملح (s)-كوينوكليدين-3-يل(2-(2-(4-فلوروفينيل)ثيازول-4-يل)بروبان-2-يل)كارباميت |
| WO2014183873A1 (en) * | 2013-05-14 | 2014-11-20 | Centogene Ag | Method for the diagnosis of niemann-pick disease |
| PL3004896T3 (pl) * | 2013-06-07 | 2020-04-30 | Genzyme Corporation | Marker zaburzeń kwaśnej spfingomielinazy i jego zastosowania |
| JP6984854B2 (ja) * | 2015-10-08 | 2021-12-22 | 国立大学法人千葉大学 | ニーマン・ピック病c型を予防または治療するための医薬組成物 |
| US10569016B2 (en) | 2015-12-29 | 2020-02-25 | Tandem Diabetes Care, Inc. | System and method for switching between closed loop and open loop control of an ambulatory infusion pump |
| SG11202001544VA (en) * | 2017-08-24 | 2020-03-30 | Sanofi Sa | Treatment of abnormal bone conditions in acid sphingomyelinase deficiency patients |
| FR3070383B1 (fr) | 2017-08-31 | 2021-08-20 | Solable | Appareil de traitement d'eau |
| BR112020005271A2 (pt) | 2017-10-02 | 2020-09-15 | Denali Therapeutics Inc. | proteína, polipeptídeo, polinucleotídeo, vetor, célula hospedeira, método para produção de um polipeptídeo, métodos de tratamento de um distúrbio, de diminuição do acúmulo, de monitoramento, de transporte de um agente e de tratamento de um lsd e composição farmacêutica |
| UY38238A (es) * | 2018-05-25 | 2019-12-31 | Genzyme Corp | Composiciones farmacéuticas para el tratamiento de la deficiencia de esfingomielinasa ácida |
| TW202142236A (zh) | 2020-02-03 | 2021-11-16 | 美商健臻公司 | 用於治療與溶體儲積症相關的神經症狀之方法 |
| AU2021311131A1 (en) | 2020-07-24 | 2023-03-23 | Genzyme Corporation | Pharmaceutical compositions comprising venglustat |
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| KR102094253B1 (ko) * | 2009-08-28 | 2020-03-31 | 이칸 스쿨 오브 메디슨 엣 마운트 시나이 | 산성 스핑고미엘리나제 결핍을 치료하기 위한 투여량 단계적 증가 효소 대체 요법 |
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| SG11202001544VA (en) * | 2017-08-24 | 2020-03-30 | Sanofi Sa | Treatment of abnormal bone conditions in acid sphingomyelinase deficiency patients |
-
2010
- 2010-08-28 KR KR1020127005293A patent/KR102094253B1/ko active Active
- 2010-08-28 CL CL2012000495A patent/CL2012000495A1/es unknown
- 2010-08-28 HU HUE10812683A patent/HUE046598T2/hu unknown
- 2010-08-28 RU RU2015145255A patent/RU2731616C2/ru active
- 2010-08-28 TR TR2019/11293T patent/TR201911293T4/tr unknown
- 2010-08-28 MX MX2017009975A patent/MX392330B/es unknown
- 2010-08-28 PL PL18199358T patent/PL3482767T3/pl unknown
- 2010-08-28 EP EP18199358.5A patent/EP3482767B1/en active Active
- 2010-08-28 DK DK10812683.0T patent/DK2470199T3/da active
- 2010-08-28 BR BR112012004377A patent/BR112012004377A2/pt not_active Application Discontinuation
- 2010-08-28 LT LTEP18199358.5T patent/LT3482767T/lt unknown
- 2010-08-28 IL IL314807A patent/IL314807A/en unknown
- 2010-08-28 PT PT212006951T patent/PT3998078T/pt unknown
- 2010-08-28 CN CN201610404696.3A patent/CN105999238A/zh active Pending
- 2010-08-28 ES ES10812683T patent/ES2738859T3/es active Active
- 2010-08-28 KR KR1020227023712A patent/KR102608551B1/ko active Active
- 2010-08-28 CA CA2773133A patent/CA2773133A1/en active Pending
- 2010-08-28 SI SI201031915T patent/SI2470199T1/sl unknown
- 2010-08-28 LT LTEP10812683.0T patent/LT2470199T/lt unknown
- 2010-08-28 WO PCT/US2010/047057 patent/WO2011025996A2/en not_active Ceased
- 2010-08-28 HR HRP20191385 patent/HRP20191385T1/hr unknown
- 2010-08-28 CN CN2010800439108A patent/CN102596232A/zh active Pending
- 2010-08-28 HR HRP20211992TT patent/HRP20211992T1/hr unknown
- 2010-08-28 SI SI201032100T patent/SI3482767T1/sl unknown
- 2010-08-28 FI FIEP21200695.1T patent/FI3998078T3/fi active
- 2010-08-28 MX MX2012002449A patent/MX356873B/es active IP Right Grant
- 2010-08-28 ES ES18199358T patent/ES2902229T3/es active Active
- 2010-08-28 AU AU2010286550A patent/AU2010286550B2/en active Active
- 2010-08-28 DK DK18199358.5T patent/DK3482767T3/da active
- 2010-08-28 HU HUE18199358A patent/HUE057440T2/hu unknown
- 2010-08-28 IL IL300625A patent/IL300625B2/en unknown
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