LT2470199T - Pakaitinė fermento terapija su dozės didinimu, skirta rūgštinės sfingomielinazės nepakankamumo gydymui - Google Patents
Pakaitinė fermento terapija su dozės didinimu, skirta rūgštinės sfingomielinazės nepakankamumo gydymuiInfo
- Publication number
- LT2470199T LT2470199T LTEP10812683.0T LT10812683T LT2470199T LT 2470199 T LT2470199 T LT 2470199T LT 10812683 T LT10812683 T LT 10812683T LT 2470199 T LT2470199 T LT 2470199T
- Authority
- LT
- Lithuania
- Prior art keywords
- replacement therapy
- dose escalation
- enzyme replacement
- acid sphingomyelinase
- treating acid
- Prior art date
Links
- 208000034012 Acid sphingomyelinase deficiency Diseases 0.000 title 1
- 238000002641 enzyme replacement therapy Methods 0.000 title 1
Classifications
-
- 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)
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 |
|---|---|
| LT2470199T true LT2470199T (lt) | 2019-10-10 |
Family
ID=43625265
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| LTEP10812683.0T LT2470199T (lt) | 2009-08-28 | 2010-08-28 | Pakaitinė fermento terapija su dozės didinimu, skirta rūgštinės sfingomielinazės nepakankamumo gydymui |
| LTEP18199358.5T LT3482767T (lt) | 2009-08-28 | 2010-08-28 | Didinamos dozės fermentų pakaitinė terapija, skirta rūgštinės sfingomielinazės nepakankamumui gydyti |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| LTEP18199358.5T LT3482767T (lt) | 2009-08-28 | 2010-08-28 | Didinamos dozės fermentų pakaitinė terapija, skirta rūgštinės sfingomielinazės nepakankamumui gydyti |
Country Status (26)
| Country | Link |
|---|---|
| US (9) | US8349319B2 (lt) |
| EP (3) | EP3482767B1 (lt) |
| JP (7) | JP5955219B2 (lt) |
| KR (5) | KR20200034828A (lt) |
| CN (3) | CN105999238A (lt) |
| AU (5) | AU2010286550B2 (lt) |
| BR (1) | BR112012004377A2 (lt) |
| CA (1) | CA2773133A1 (lt) |
| CL (1) | CL2012000495A1 (lt) |
| CY (1) | CY1122128T1 (lt) |
| DK (2) | DK3482767T3 (lt) |
| ES (2) | ES2902229T3 (lt) |
| FR (1) | FR22C1062I2 (lt) |
| HR (2) | HRP20211992T1 (lt) |
| HU (3) | HUE046598T2 (lt) |
| IL (3) | IL300625A (lt) |
| LT (2) | LT2470199T (lt) |
| MX (1) | MX356873B (lt) |
| NL (1) | NL301211I2 (lt) |
| PL (2) | PL3482767T3 (lt) |
| PT (2) | PT3482767T (lt) |
| RU (3) | RU2731616C2 (lt) |
| SI (2) | SI2470199T1 (lt) |
| TR (1) | TR201911293T4 (lt) |
| WO (1) | WO2011025996A2 (lt) |
| ZA (2) | ZA201201435B (lt) |
Families Citing this family (23)
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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 |
| CN107080747B (zh) | 2011-03-18 | 2021-02-26 | 建新公司 | 葡萄糖基神经酰胺合酶抑制剂 |
| DK2753346T3 (da) | 2011-09-07 | 2020-05-25 | Sinai School Medicine | Ceramidase og celledifferentiering |
| MX359328B (es) * | 2011-11-15 | 2018-09-25 | Centogene Ag | Metodo para el diagnostico de enfermedad de niemann-pick. |
| EP3679923B1 (en) | 2012-06-01 | 2021-08-04 | Icahn School of Medicine at Mount Sinai | Ceramide levels in the treatment and prevention of infections |
| JOP20130273B1 (ar) | 2012-09-11 | 2021-08-17 | Genzyme Corp | مثبطات انزيم (سينثاز) غلوكوسيل سيراميد |
| JP6832158B2 (ja) * | 2013-03-14 | 2021-02-24 | アイカーン スクール オブ メディシン アット マウント サイナイ | 治療用酸性セラミダーゼ組成物ならびにそれを作製および使用する方法 |
| 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 |
| ES2731920T3 (es) * | 2013-05-14 | 2019-11-19 | Centogene Ag | Método para el diagnóstico de la enfermedad de Niemann-Pick |
| HUE047863T2 (hu) * | 2013-06-07 | 2020-05-28 | Genzyme Corp | Marker savas szfingomielináz rendellenességekhez és alkalmazásai |
| 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 |
| 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 |
| US11857512B2 (en) | 2020-07-24 | 2024-01-02 | Genzyme Corporation | Pharmaceutical compositions comprising 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 |
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2010
- 2010-08-28 PT PT181993585T patent/PT3482767T/pt unknown
- 2010-08-28 SI SI201031915T patent/SI2470199T1/sl unknown
- 2010-08-28 PL PL18199358T patent/PL3482767T3/pl unknown
- 2010-08-28 KR KR1020207008342A patent/KR20200034828A/ko not_active IP Right Cessation
- 2010-08-28 EP EP18199358.5A patent/EP3482767B1/en active Active
- 2010-08-28 RU RU2015145255A patent/RU2731616C2/ru active
- 2010-08-28 DK DK18199358.5T patent/DK3482767T3/da active
- 2010-08-28 CN CN201610404696.3A patent/CN105999238A/zh active Pending
- 2010-08-28 BR BR112012004377A patent/BR112012004377A2/pt not_active Application Discontinuation
- 2010-08-28 WO PCT/US2010/047057 patent/WO2011025996A2/en active Application Filing
- 2010-08-28 KR KR1020217026374A patent/KR102608546B1/ko active IP Right Grant
- 2010-08-28 US US12/870,790 patent/US8349319B2/en active Active
- 2010-08-28 HU HUE10812683A patent/HUE046598T2/hu unknown
- 2010-08-28 EP EP21200695.1A patent/EP3998078A1/en active Pending
- 2010-08-28 HU HUE18199358A patent/HUE057440T2/hu unknown
- 2010-08-28 HR HRP20211992TT patent/HRP20211992T1/hr unknown
- 2010-08-28 CN CN2010800439108A patent/CN102596232A/zh active Pending
- 2010-08-28 IL IL300625A patent/IL300625A/en unknown
- 2010-08-28 CN CN201610403228.4A patent/CN106047835A/zh active Pending
- 2010-08-28 AU AU2010286550A patent/AU2010286550B2/en active Active
- 2010-08-28 JP JP2012527054A patent/JP5955219B2/ja active Active
- 2010-08-28 ES ES18199358T patent/ES2902229T3/es active Active
- 2010-08-28 KR KR1020237040891A patent/KR20230164773A/ko active Application Filing
- 2010-08-28 PT PT10812683T patent/PT2470199T/pt unknown
- 2010-08-28 MX MX2012002449A patent/MX356873B/es active IP Right Grant
- 2010-08-28 RU RU2012111823/15A patent/RU2569744C2/ru active
- 2010-08-28 KR KR1020227023712A patent/KR102608551B1/ko active IP Right Grant
- 2010-08-28 KR KR1020127005293A patent/KR102094253B1/ko active IP Right Grant
- 2010-08-28 EP EP10812683.0A patent/EP2470199B1/en active Active
- 2010-08-28 ES ES10812683T patent/ES2738859T3/es active Active
- 2010-08-28 SI SI201032100T patent/SI3482767T1/sl unknown
- 2010-08-28 DK DK10812683.0T patent/DK2470199T3/da active
- 2010-08-28 LT LTEP10812683.0T patent/LT2470199T/lt unknown
- 2010-08-28 TR TR2019/11293T patent/TR201911293T4/tr unknown
- 2010-08-28 PL PL10812683T patent/PL2470199T3/pl unknown
- 2010-08-28 CA CA2773133A patent/CA2773133A1/en active Pending
- 2010-08-28 LT LTEP18199358.5T patent/LT3482767T/lt unknown
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2012
- 2012-02-26 IL IL218324A patent/IL218324B/en active IP Right Grant
- 2012-02-27 ZA ZA2012/01435A patent/ZA201201435B/en unknown
- 2012-02-27 CL CL2012000495A patent/CL2012000495A1/es unknown
- 2012-11-16 US US13/679,623 patent/US8709408B2/en active Active
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2018
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