MX2012002449A - Terapia de reemplazo de enzimas con aumento escalonado de la dosis para tratar la deficiencia de esfingomielinasa acida. - Google Patents
Terapia de reemplazo de enzimas con aumento escalonado de la dosis para tratar la deficiencia de esfingomielinasa acida.Info
- Publication number
- MX2012002449A MX2012002449A MX2012002449A MX2012002449A MX2012002449A MX 2012002449 A MX2012002449 A MX 2012002449A MX 2012002449 A MX2012002449 A MX 2012002449A MX 2012002449 A MX2012002449 A MX 2012002449A MX 2012002449 A MX2012002449 A MX 2012002449A
- Authority
- MX
- Mexico
- Prior art keywords
- acid sphingomyelinase
- replacement therapy
- dose escalation
- enzyme replacement
- treating acid
- Prior art date
Links
- 208000034012 Acid sphingomyelinase deficiency Diseases 0.000 title abstract 3
- 238000002641 enzyme replacement therapy Methods 0.000 title abstract 2
- 208000014060 Niemann-Pick disease Diseases 0.000 abstract 3
- 108010061312 Sphingomyelin Phosphodiesterase Proteins 0.000 abstract 2
- 102000010126 acid sphingomyelin phosphodiesterase activity proteins Human genes 0.000 abstract 2
- 208000008457 Neurologic Manifestations 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 con la terapia de reemplazo de enzimas con aumento escalonado de la dosis que utiliza la esfingomielinasa ácida (ASM) para el tratamiento de sujetos humanos con deficiencia de esfingomielinasa ácida (ASMD) y, en particular, pacientes con manifestaciones no neurológicas de la enfermedad de Niemann Pick (NPD) y, en determinadas aplicaciones, con NPD tipo B.
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 (2)
Publication Number | Publication Date |
---|---|
MX2012002449A true MX2012002449A (es) | 2012-03-14 |
MX356873B MX356873B (es) | 2018-06-18 |
Family
ID=43625265
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MX2012002449A MX356873B (es) | 2009-08-28 | 2010-08-28 | Uso farmacéutico de una esfingomielinasa ácida (asm) con aumento escalonada de la dosis para tratar la deficiencia de esfingomielinasa ácida. |
Country Status (26)
Country | Link |
---|---|
US (9) | US8349319B2 (es) |
EP (3) | EP3998078A1 (es) |
JP (7) | JP5955219B2 (es) |
KR (5) | KR20230164773A (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) | DK2470199T3 (es) |
ES (2) | ES2738859T3 (es) |
FR (1) | FR22C1062I2 (es) |
HR (2) | HRP20211992T1 (es) |
HU (3) | HUE057440T2 (es) |
IL (3) | IL300625A (es) |
LT (2) | LT3482767T (es) |
MX (1) | MX356873B (es) |
NL (1) | NL301211I2 (es) |
PL (2) | PL2470199T3 (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 (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 |
WO2008154198A1 (en) | 2007-06-06 | 2008-12-18 | Genzyme Corporation | Gene therapy for lysosomal storage diseases |
IL300625A (en) * | 2009-08-28 | 2023-04-01 | Icahn School Med Mount Sinai | Increasing dose replacement enzyme for the treatment of acid sphingomyelinase deficiency |
US8882701B2 (en) * | 2009-12-04 | 2014-11-11 | Smiths Medical Asd, Inc. | Advanced step therapy delivery for an ambulatory infusion pump and system |
JP2013528379A (ja) | 2010-05-21 | 2013-07-11 | オームクス コーポレーション | Psa酵素活性アッセイによる癌の検出 |
MX365245B (es) | 2011-03-18 | 2019-05-28 | Genzyme Corp | Inhibidores de glucosilceramida sintasa. |
EP3741384A1 (en) | 2011-09-07 | 2020-11-25 | Mount Sinai School Of Medicine | Ceramidase and cell differentiation |
EP2780716B1 (en) * | 2011-11-15 | 2019-07-31 | Centogene AG | Method for the diagnosis of niemann-pick disease |
CN104582770B (zh) | 2012-06-01 | 2019-01-29 | 西奈山伊坎医学院 | 在感染的治疗和预防中的神经酰胺水平 |
MA37975B2 (fr) | 2012-09-11 | 2021-03-31 | Genzyme Corp | Inhibiteurs de synthase de glucosylcéramide |
PT2968479T (pt) | 2013-03-14 | 2019-08-07 | Icahn School Med Mount Sinai | Composições terapêuticas de ceramidase ácida e métodos de fabrico e utilização |
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 |
SG10201709925SA (en) * | 2013-06-07 | 2017-12-28 | 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 |
CA3073648A1 (en) | 2017-08-24 | 2019-02-28 | Sanofi | Treatment of abnormal bone conditions in acid sphingomyelinase deficiency patients |
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 |
WO2022018695A1 (en) | 2020-07-24 | 2022-01-27 | Genzyme Corporation | Pharmaceutical compositions comprising venglustat |
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2010
- 2010-08-28 IL IL300625A patent/IL300625A/en unknown
- 2010-08-28 MX MX2012002449A patent/MX356873B/es active IP Right Grant
- 2010-08-28 LT LTEP18199358.5T patent/LT3482767T/lt unknown
- 2010-08-28 EP EP21200695.1A patent/EP3998078A1/en active Pending
- 2010-08-28 PL PL10812683T patent/PL2470199T3/pl unknown
- 2010-08-28 EP EP18199358.5A patent/EP3482767B1/en active Active
- 2010-08-28 CA CA2773133A patent/CA2773133A1/en active Pending
- 2010-08-28 CN CN201610403228.4A patent/CN106047835A/zh active Pending
- 2010-08-28 PT PT181993585T patent/PT3482767T/pt unknown
- 2010-08-28 KR KR1020237040891A patent/KR20230164773A/ko active Application Filing
- 2010-08-28 JP JP2012527054A patent/JP5955219B2/ja active Active
- 2010-08-28 HU HUE18199358A patent/HUE057440T2/hu unknown
- 2010-08-28 EP EP10812683.0A patent/EP2470199B1/en active Active
- 2010-08-28 US US12/870,790 patent/US8349319B2/en active Active
- 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 HU HUE10812683A patent/HUE046598T2/hu unknown
- 2010-08-28 ES ES10812683T patent/ES2738859T3/es active Active
- 2010-08-28 DK DK10812683.0T patent/DK2470199T3/da active
- 2010-08-28 PT PT10812683T patent/PT2470199T/pt unknown
- 2010-08-28 KR KR1020207008342A patent/KR20200034828A/ko not_active IP Right Cessation
- 2010-08-28 SI SI201032100T patent/SI3482767T1/sl unknown
- 2010-08-28 CN CN2010800439108A patent/CN102596232A/zh active Pending
- 2010-08-28 RU RU2015145255A patent/RU2731616C2/ru active
- 2010-08-28 LT LTEP10812683.0T patent/LT2470199T/lt unknown
- 2010-08-28 SI SI201031915T patent/SI2470199T1/sl unknown
- 2010-08-28 WO PCT/US2010/047057 patent/WO2011025996A2/en active Application Filing
- 2010-08-28 DK DK18199358.5T patent/DK3482767T3/da active
- 2010-08-28 TR TR2019/11293T patent/TR201911293T4/tr unknown
- 2010-08-28 PL PL18199358T patent/PL3482767T3/pl unknown
- 2010-08-28 BR BR112012004377A patent/BR112012004377A2/pt not_active Application Discontinuation
- 2010-08-28 ES ES18199358T patent/ES2902229T3/es active Active
- 2010-08-28 KR KR1020217026374A patent/KR102608546B1/ko active IP Right Grant
- 2010-08-28 HR HRP20211992TT patent/HRP20211992T1/hr unknown
- 2010-08-28 AU AU2010286550A patent/AU2010286550B2/en active Active
- 2010-08-28 CN CN201610404696.3A patent/CN105999238A/zh active Pending
- 2010-08-28 KR KR1020127005293A patent/KR102094253B1/ko active IP Right Grant
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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
- 2012-11-16 US US13/679,583 patent/US8658162B2/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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2018
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