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)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Biomedical Technology (AREA)
- Genetics & Genomics (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Gastroenterology & Hepatology (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Epidemiology (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Obesity (AREA)
- Hematology (AREA)
- Diabetes (AREA)
- Hospice & Palliative Care (AREA)
- Psychiatry (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 (1)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US23811309P | 2009-08-28 | 2009-08-28 |
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 | 2012-02-27 | Producto farmacéutico para la citoreducción de sustrato de esfingomielina acumulada y tratar deficiencia de esfingo mielinasa acida (asmd). |
Country Status (27)
Country | Link |
---|---|
US (9) | US8349319B2 (es) |
EP (3) | EP3998078A1 (es) |
JP (7) | JP5955219B2 (es) |
KR (5) | KR102094253B1 (es) |
CN (3) | CN106047835A (es) |
AU (5) | AU2010286550B2 (es) |
BR (1) | BR112012004377A2 (es) |
CA (1) | CA2773133A1 (es) |
CL (1) | CL2012000495A1 (es) |
CY (1) | CY1122128T1 (es) |
DK (2) | DK3482767T3 (es) |
ES (2) | ES2738859T3 (es) |
FR (1) | FR22C1062I2 (es) |
HR (2) | HRP20211992T1 (es) |
HU (3) | HUE046598T2 (es) |
IL (4) | IL300625B1 (es) |
LT (2) | LT3482767T (es) |
MX (1) | MX356873B (es) |
NL (1) | NL301211I2 (es) |
NO (1) | NO2022054I1 (es) |
PL (2) | PL3482767T3 (es) |
PT (2) | PT3482767T (es) |
RU (3) | RU2569744C2 (es) |
SI (2) | SI3482767T1 (es) |
TR (1) | TR201911293T4 (es) |
WO (1) | WO2011025996A2 (es) |
ZA (2) | ZA201201435B (es) |
Families Citing this family (24)
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US8889127B2 (en) | 2004-07-01 | 2014-11-18 | Icahn School Of Medicine At Mount Sinai | 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 |
ES2905616T3 (es) | 2007-06-06 | 2022-04-11 | Genzyme Corp | Terapia génica para enfermedades de almacenamiento lisosómico |
PT3482767T (pt) * | 2009-08-28 | 2021-12-29 | Icahn School Med Mount Sinai | Terapia de substituição enzimática com escaladas de dose para tratamento de deficência da esfingomielinase ácida |
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 |
DK2685986T3 (da) | 2011-03-18 | 2020-03-02 | Genzyme Corp | Glucosylceramidsyntasehæmmer |
HUE051021T2 (hu) | 2011-09-07 | 2021-01-28 | Sinai School Medicine | Ceramidáz és sejtek differenciálódása |
MX359328B (es) | 2011-11-15 | 2018-09-25 | Centogene Ag | Metodo para el diagnostico de enfermedad de niemann-pick. |
DK2854910T3 (da) | 2012-06-01 | 2020-05-04 | Icahn School Med Mount Sinai | Ceramidniveauer i behandling og forebyggelse af infektioner |
MA37975B2 (fr) | 2012-09-11 | 2021-03-31 | Genzyme Corp | Inhibiteurs de synthase de glucosylcéramide |
CA2905449A1 (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 |
CA2912274A1 (en) * | 2013-05-14 | 2014-11-20 | Centogene Ag | Method for the diagnosis of niemann-pick disease |
EA035342B1 (ru) * | 2013-06-07 | 2020-05-29 | Джензим Корпорейшн | Способы контроля лечения пациента, имеющего ассоциированное с кислой сфингомиелиназой (asm) нарушение |
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 |
EP3672622B1 (en) * | 2017-08-24 | 2023-11-08 | Genzyme Corporation | Recombinant human acid sphingomyelinase for use in treating abnormal bone conditions in acid sphingomyelinase deficiency patients |
CA3076369A1 (en) | 2017-10-02 | 2019-04-11 | Denali Therapeutics Inc. | Fusion proteins comprising enzyme replacement therapy enzymes |
UY38238A (es) * | 2018-05-25 | 2019-12-31 | Genzyme Corp | Composiciones farmacéuticas para el tratamiento de la deficiencia de esfingomielinasa ácida |
AU2021215396A1 (en) | 2020-02-03 | 2022-09-29 | Genzyme Corporation | Methods for treating neurological symptoms associated with lysosomal storage diseases |
IL300090A (en) | 2020-07-24 | 2023-03-01 | Genzyme Corp | Pharmaceutical preparations containing VENGLUSTAT |
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PT3482767T (pt) * | 2009-08-28 | 2021-12-29 | Icahn School Med Mount Sinai | Terapia de substituição enzimática com escaladas de dose para tratamento de deficência da esfingomielinase ácida |
CN103764824B (zh) * | 2011-06-20 | 2018-04-24 | 西奈山医学院 | 粘多糖贮积症和其它溶酶体病症的抗TNF-α疗法 |
EA035342B1 (ru) * | 2013-06-07 | 2020-05-29 | Джензим Корпорейшн | Способы контроля лечения пациента, имеющего ассоциированное с кислой сфингомиелиназой (asm) нарушение |
EP3672622B1 (en) * | 2017-08-24 | 2023-11-08 | Genzyme Corporation | Recombinant human acid sphingomyelinase for use in treating abnormal bone conditions in acid sphingomyelinase deficiency patients |
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2010
- 2010-08-28 PT PT181993585T patent/PT3482767T/pt unknown
- 2010-08-28 CN CN201610403228.4A patent/CN106047835A/zh active Pending
- 2010-08-28 HU HUE10812683A patent/HUE046598T2/hu unknown
- 2010-08-28 PL PL18199358T patent/PL3482767T3/pl unknown
- 2010-08-28 KR KR1020127005293A patent/KR102094253B1/ko active IP Right Grant
- 2010-08-28 IL IL300625A patent/IL300625B1/en unknown
- 2010-08-28 HR HRP20211992TT patent/HRP20211992T1/hr unknown
- 2010-08-28 AU AU2010286550A patent/AU2010286550B2/en active Active
- 2010-08-28 EP EP21200695.1A patent/EP3998078A1/en active Pending
- 2010-08-28 WO PCT/US2010/047057 patent/WO2011025996A2/en active Application Filing
- 2010-08-28 ES ES10812683T patent/ES2738859T3/es active Active
- 2010-08-28 RU RU2012111823/15A patent/RU2569744C2/ru active
- 2010-08-28 RU RU2015145255A patent/RU2731616C2/ru active
- 2010-08-28 ES ES18199358T patent/ES2902229T3/es active Active
- 2010-08-28 CA CA2773133A patent/CA2773133A1/en active Pending
- 2010-08-28 SI SI201032100T patent/SI3482767T1/sl unknown
- 2010-08-28 JP JP2012527054A patent/JP5955219B2/ja active Active
- 2010-08-28 KR KR1020237040891A patent/KR20230164773A/ko active Application Filing
- 2010-08-28 DK DK18199358.5T patent/DK3482767T3/da active
- 2010-08-28 PL PL10812683T patent/PL2470199T3/pl unknown
- 2010-08-28 EP EP10812683.0A patent/EP2470199B1/en active Active
- 2010-08-28 DK DK10812683.0T patent/DK2470199T3/da active
- 2010-08-28 US US12/870,790 patent/US8349319B2/en active Active
- 2010-08-28 KR KR1020207008342A patent/KR20200034828A/ko not_active IP Right Cessation
- 2010-08-28 PT PT10812683T patent/PT2470199T/pt unknown
- 2010-08-28 BR BR112012004377A patent/BR112012004377A2/pt not_active Application Discontinuation
- 2010-08-28 MX MX2012002449A patent/MX356873B/es active IP Right Grant
- 2010-08-28 KR KR1020227023712A patent/KR102608551B1/ko active IP Right Grant
- 2010-08-28 KR KR1020217026374A patent/KR102608546B1/ko active IP Right Grant
- 2010-08-28 CN CN201610404696.3A patent/CN105999238A/zh active Pending
- 2010-08-28 CN CN2010800439108A patent/CN102596232A/zh active Pending
- 2010-08-28 LT LTEP18199358.5T patent/LT3482767T/lt unknown
- 2010-08-28 TR TR2019/11293T patent/TR201911293T4/tr unknown
- 2010-08-28 EP EP18199358.5A patent/EP3482767B1/en active Active
- 2010-08-28 SI SI201031915T patent/SI2470199T1/sl unknown
- 2010-08-28 IL IL314807A patent/IL314807A/en unknown
- 2010-08-28 HU HUE18199358A patent/HUE057440T2/hu unknown
- 2010-08-28 LT LTEP10812683.0T patent/LT2470199T/lt unknown
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2012
- 2012-02-26 IL IL218324A patent/IL218324B/en active IP Right Grant
- 2012-02-27 CL CL2012000495A patent/CL2012000495A1/es unknown
- 2012-02-27 ZA ZA2012/01435A patent/ZA201201435B/en unknown
- 2012-11-16 US US13/679,583 patent/US8658162B2/en active Active
- 2012-11-16 US US13/679,623 patent/US8709408B2/en active Active
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2014
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2018
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