PL2470199T3 - Enzymatyczna terapia zastępcza ze zwiększaniem dawki do leczenia niedoboru kwaśnej sfingomielinazy - Google Patents
Enzymatyczna terapia zastępcza ze zwiększaniem dawki do leczenia niedoboru kwaśnej sfingomielinazyInfo
- Publication number
- PL2470199T3 PL2470199T3 PL10812683T PL10812683T PL2470199T3 PL 2470199 T3 PL2470199 T3 PL 2470199T3 PL 10812683 T PL10812683 T PL 10812683T PL 10812683 T PL10812683 T PL 10812683T PL 2470199 T3 PL2470199 T3 PL 2470199T3
- Authority
- PL
- Poland
- 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)
- 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)
- Neurology (AREA)
- Neurosurgery (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)
- 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 (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US23811309P | 2009-08-28 | 2009-08-28 | |
EP10812683.0A EP2470199B1 (en) | 2009-08-28 | 2010-08-28 | Dose escalation enzyme replacement therapy for treating acid sphingomyelinase deficiency |
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 |
---|---|
PL2470199T3 true PL2470199T3 (pl) | 2019-11-29 |
Family
ID=43625265
Family Applications (2)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
PL10812683T PL2470199T3 (pl) | 2009-08-28 | 2010-08-28 | Enzymatyczna terapia zastępcza ze zwiększaniem dawki do leczenia niedoboru kwaśnej sfingomielinazy |
PL18199358T PL3482767T3 (pl) | 2009-08-28 | 2010-08-28 | Enzymatyczna terapia zastępcza ze zwiększaniem dawki do leczenia niedoboru kwaśnej sfingomielinazy |
Family Applications After (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
PL18199358T PL3482767T3 (pl) | 2009-08-28 | 2010-08-28 | Enzymatyczna terapia zastępcza ze zwiększaniem dawki do leczenia niedoboru kwaśnej sfingomielinazy |
Country Status (26)
Country | Link |
---|---|
US (9) | US8349319B2 (pl) |
EP (3) | EP2470199B1 (pl) |
JP (7) | JP5955219B2 (pl) |
KR (5) | KR20230164773A (pl) |
CN (3) | CN105999238A (pl) |
AU (5) | AU2010286550B2 (pl) |
BR (1) | BR112012004377A2 (pl) |
CA (1) | CA2773133A1 (pl) |
CL (1) | CL2012000495A1 (pl) |
CY (1) | CY1122128T1 (pl) |
DK (2) | DK2470199T3 (pl) |
ES (2) | ES2902229T3 (pl) |
FR (1) | FR22C1062I2 (pl) |
HR (2) | HRP20211992T1 (pl) |
HU (3) | HUE046598T2 (pl) |
IL (3) | IL300625A (pl) |
LT (2) | LT2470199T (pl) |
MX (1) | MX356873B (pl) |
NL (1) | NL301211I2 (pl) |
PL (2) | PL2470199T3 (pl) |
PT (2) | PT2470199T (pl) |
RU (3) | RU2731616C2 (pl) |
SI (2) | SI2470199T1 (pl) |
TR (1) | TR201911293T4 (pl) |
WO (1) | WO2011025996A2 (pl) |
ZA (2) | ZA201201435B (pl) |
Families Citing this family (23)
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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 |
EP2158322B1 (en) | 2007-06-06 | 2017-05-03 | Genzyme Corporation | Gene therapy for lysosomal storage diseases |
KR20230164773A (ko) * | 2009-08-28 | 2023-12-04 | 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 |
CA2798812A1 (en) | 2010-05-21 | 2011-11-24 | Ohmx Corporation | Detection of cancer by assaying psa enzymatic activity |
TWI546301B (zh) | 2011-03-18 | 2016-08-21 | 健臻公司 | 葡萄糖神經醯胺合成酶抑制劑 |
US10350277B2 (en) | 2011-09-07 | 2019-07-16 | Icahn School Of Medicine At Mount Sinai | Ceramidase and cell differentiation |
DK2780716T3 (da) * | 2011-11-15 | 2019-10-14 | Centogene Ag | Fremgangsmåde til diagnose af Niemann-Pick sygdom |
PT2854910T (pt) | 2012-06-01 | 2020-05-06 | Yeda Res & Dev | Níveis de ceramida no tratamento e prevenção de infeções |
JOP20130273B1 (ar) | 2012-09-11 | 2021-08-17 | Genzyme Corp | مثبطات انزيم (سينثاز) غلوكوسيل سيراميد |
ES2739811T3 (es) * | 2013-03-14 | 2020-02-04 | Icahn School Med Mount Sinai | Composiciones terapéuticas de ceramidasa ácida y métodos de fabricación y uso de ellas |
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 |
SG11201509622WA (en) * | 2013-06-07 | 2015-12-30 | Genzyme Corp | Marker for acid sphingomyelinase disorders and uses thereof |
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 |
MX2020002106A (es) * | 2017-08-24 | 2020-07-14 | Sanofi Sa | Tratamiento de afecciones oseas anormales en pacientes con deficiencia de esfingomielinasa acida. |
JP2021500857A (ja) | 2017-10-02 | 2021-01-14 | デナリ セラピューティクス インコーポレイテッドDenali Therapeutics Inc. | 酵素補充療法用酵素を含む融合タンパク質 |
UY38238A (es) | 2018-05-25 | 2019-12-31 | Genzyme Corp | Composiciones farmacéuticas para el tratamiento de la deficiencia de esfingomielinasa ácida |
EP4185267A1 (en) | 2020-07-24 | 2023-05-31 | Genzyme Corporation | Pharmaceutical compositions comprising venglustat |
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KR20230164773A (ko) * | 2009-08-28 | 2023-12-04 | 이칸 스쿨 오브 메디슨 엣 마운트 시나이 | 산성 스핑고미엘리나제 결핍을 치료하기 위한 투여량 단계적 증가 효소 대체 요법 |
ES2664844T3 (es) * | 2011-06-20 | 2018-04-23 | Mount Sinai School Of Medicine | Tratamiento dirigido contra TNF para las mucopolisacaridosis y otros trastornos lisosómicos |
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2010
- 2010-08-28 KR KR1020237040891A patent/KR20230164773A/ko active Application Filing
- 2010-08-28 LT LTEP10812683.0T patent/LT2470199T/lt unknown
- 2010-08-28 DK DK10812683.0T patent/DK2470199T3/da active
- 2010-08-28 RU RU2015145255A patent/RU2731616C2/ru active
- 2010-08-28 CN CN201610404696.3A patent/CN105999238A/zh active Pending
- 2010-08-28 EP EP10812683.0A patent/EP2470199B1/en active Active
- 2010-08-28 CA CA2773133A patent/CA2773133A1/en active Pending
- 2010-08-28 ES ES18199358T patent/ES2902229T3/es active Active
- 2010-08-28 TR TR2019/11293T patent/TR201911293T4/tr unknown
- 2010-08-28 PT PT10812683T patent/PT2470199T/pt unknown
- 2010-08-28 US US12/870,790 patent/US8349319B2/en active Active
- 2010-08-28 WO PCT/US2010/047057 patent/WO2011025996A2/en active Application Filing
- 2010-08-28 SI SI201031915T patent/SI2470199T1/sl unknown
- 2010-08-28 PT PT181993585T patent/PT3482767T/pt unknown
- 2010-08-28 HU HUE10812683A patent/HUE046598T2/hu unknown
- 2010-08-28 CN CN201610403228.4A patent/CN106047835A/zh active Pending
- 2010-08-28 KR KR1020207008342A patent/KR20200034828A/ko not_active IP Right Cessation
- 2010-08-28 LT LTEP18199358.5T patent/LT3482767T/lt unknown
- 2010-08-28 IL IL300625A patent/IL300625A/en unknown
- 2010-08-28 ES ES10812683T patent/ES2738859T3/es active Active
- 2010-08-28 RU RU2012111823/15A patent/RU2569744C2/ru active
- 2010-08-28 CN CN2010800439108A patent/CN102596232A/zh active Pending
- 2010-08-28 PL PL10812683T patent/PL2470199T3/pl unknown
- 2010-08-28 MX MX2012002449A patent/MX356873B/es active IP Right Grant
- 2010-08-28 JP JP2012527054A patent/JP5955219B2/ja active Active
- 2010-08-28 HR HRP20211992TT patent/HRP20211992T1/hr unknown
- 2010-08-28 KR KR1020127005293A patent/KR102094253B1/ko active IP Right Grant
- 2010-08-28 SI SI201032100T patent/SI3482767T1/sl unknown
- 2010-08-28 PL PL18199358T patent/PL3482767T3/pl unknown
- 2010-08-28 EP EP18199358.5A patent/EP3482767B1/en active Active
- 2010-08-28 EP EP21200695.1A patent/EP3998078A1/en active Pending
- 2010-08-28 KR KR1020217026374A patent/KR102608546B1/ko active IP Right Grant
- 2010-08-28 KR KR1020227023712A patent/KR102608551B1/ko active IP Right Grant
- 2010-08-28 DK DK18199358.5T patent/DK3482767T3/da active
- 2010-08-28 HU HUE18199358A patent/HUE057440T2/hu unknown
- 2010-08-28 BR BR112012004377A patent/BR112012004377A2/pt not_active Application Discontinuation
- 2010-08-28 AU AU2010286550A patent/AU2010286550B2/en active Active
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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 US13/679,583 patent/US8658162B2/en active Active
- 2012-11-16 US US13/679,623 patent/US8709408B2/en active Active
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2014
- 2014-01-16 US US14/156,894 patent/US9114139B2/en active Active
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2015
- 2015-07-08 US US14/793,838 patent/US9655954B2/en active Active
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2016
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- 2016-03-03 JP JP2016041242A patent/JP6251307B2/ja active Active
- 2016-08-17 AU AU2016216625A patent/AU2016216625B2/en active Active
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2017
- 2017-04-12 US US15/485,604 patent/US10188705B2/en active Active
- 2017-11-24 JP JP2017226255A patent/JP2018076322A/ja active Pending
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2018
- 2018-06-12 AU AU2018204156A patent/AU2018204156B2/en active Active
- 2018-12-04 US US16/209,336 patent/US20190151419A1/en not_active Abandoned
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2019
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- 2019-07-29 CY CY20191100808T patent/CY1122128T1/el unknown
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2020
- 2020-01-17 JP JP2020005901A patent/JP6952810B2/ja active Active
- 2020-05-26 US US16/883,407 patent/US20210077593A1/en not_active Abandoned
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2021
- 2021-09-28 JP JP2021157902A patent/JP7167281B2/ja active Active
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2022
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- 2022-12-08 HU HUS2200050C patent/HUS2200050I1/hu unknown
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2023
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2024
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