FR22C1062I1 - Thérapie de remplacement enzymatique d'escalade de doses pour le traitement du déficit en sphingomyélinase acide - Google Patents
Thérapie de remplacement enzymatique d'escalade de doses pour le traitement du déficit en sphingomyélinase acideInfo
- Publication number
- FR22C1062I1 FR22C1062I1 FR22C1062C FR22C1062C FR22C1062I1 FR 22C1062 I1 FR22C1062 I1 FR 22C1062I1 FR 22C1062 C FR22C1062 C FR 22C1062C FR 22C1062 C FR22C1062 C FR 22C1062C FR 22C1062 I1 FR22C1062 I1 FR 22C1062I1
- Authority
- FR
- France
- Prior art keywords
- enzy
- treatment
- replacement therapy
- dose escalation
- acid sphingomyelinase
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Active
Links
- 208000034012 Acid sphingomyelinase deficiency Diseases 0.000 title 1
- 238000009256 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 (fr) | 2009-08-28 | 2010-08-28 | Thérapie de substitution enzymatique à doses croissantes permettant de traiter un déficit en sphingomyélinase acide |
PCT/US2010/047057 WO2011025996A2 (fr) | 2009-08-28 | 2010-08-28 | Thérapie de substitution enzymatique à doses croissantes permettant de traiter un déficit en sphingomyélinase acide |
Publications (2)
Publication Number | Publication Date |
---|---|
FR22C1062I1 true FR22C1062I1 (fr) | 2023-02-03 |
FR22C1062I2 FR22C1062I2 (fr) | 2023-12-29 |
Family
ID=43625265
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
FR22C1062C Active FR22C1062I2 (fr) | 2009-08-28 | 2022-12-15 | Thérapie de remplacement enzymatique d'escalade de doses pour le traitement du déficit en sphingomyélinase acide |
Country Status (26)
Country | Link |
---|---|
US (9) | US8349319B2 (fr) |
EP (3) | EP2470199B1 (fr) |
JP (7) | JP5955219B2 (fr) |
KR (5) | KR20230164773A (fr) |
CN (3) | CN105999238A (fr) |
AU (5) | AU2010286550B2 (fr) |
BR (1) | BR112012004377A2 (fr) |
CA (1) | CA2773133A1 (fr) |
CL (1) | CL2012000495A1 (fr) |
CY (1) | CY1122128T1 (fr) |
DK (2) | DK2470199T3 (fr) |
ES (2) | ES2902229T3 (fr) |
FR (1) | FR22C1062I2 (fr) |
HR (2) | HRP20211992T1 (fr) |
HU (3) | HUE046598T2 (fr) |
IL (3) | IL300625A (fr) |
LT (2) | LT2470199T (fr) |
MX (1) | MX356873B (fr) |
NL (1) | NL301211I2 (fr) |
PL (2) | PL2470199T3 (fr) |
PT (2) | PT2470199T (fr) |
RU (3) | RU2731616C2 (fr) |
SI (2) | SI2470199T1 (fr) |
TR (1) | TR201911293T4 (fr) |
WO (1) | WO2011025996A2 (fr) |
ZA (2) | ZA201201435B (fr) |
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WO2006007560A2 (fr) | 2004-07-01 | 2006-01-19 | University Of Pennsylvania | Remplacement de proteines ciblees pour le traitement de maladies lysosomales |
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 (fr) | 2007-06-06 | 2017-05-03 | Genzyme Corporation | Thérapie génique pour les maladies de stockage lysosomal |
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 (fr) | 2010-05-21 | 2011-11-24 | Ohmx Corporation | Detection du cancer par dosage de l'activite enzymatique du psa |
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 (fr) * | 2013-05-14 | 2014-11-20 | Centogene Ag | Procédé pour le diagnostic de la maladie de niemann-pick |
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 (fr) | 2020-07-24 | 2023-05-31 | Genzyme Corporation | Compositions pharmaceutiques comprenant du venglustat |
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2010
- 2010-08-28 KR KR1020237040891A patent/KR20230164773A/ko active Application Filing
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- 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/fr active Active
- 2010-08-28 CA CA2773133A patent/CA2773133A1/fr 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/fr active Application Filing
- 2010-08-28 SI SI201031915T patent/SI2470199T1/sl unknown
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- 2010-08-28 HU HUE10812683A patent/HUE046598T2/hu unknown
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- 2010-08-28 KR KR1020207008342A patent/KR20200034828A/ko not_active IP Right Cessation
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- 2010-08-28 EP EP18199358.5A patent/EP3482767B1/fr active Active
- 2010-08-28 EP EP21200695.1A patent/EP3998078A1/fr 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
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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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- 2016-02-26 ZA ZA2016/01362A patent/ZA201601362B/en unknown
- 2016-03-03 JP JP2016041242A patent/JP6251307B2/ja active Active
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2017
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2018
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2019
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2020
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2021
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2022
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2023
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2024
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