CA3073648A1 - Treatment of abnormal bone conditions in acid sphingomyelinase deficiency patients - Google Patents
Treatment of abnormal bone conditions in acid sphingomyelinase deficiency patients Download PDFInfo
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- CA3073648A1 CA3073648A1 CA3073648A CA3073648A CA3073648A1 CA 3073648 A1 CA3073648 A1 CA 3073648A1 CA 3073648 A CA3073648 A CA 3073648A CA 3073648 A CA3073648 A CA 3073648A CA 3073648 A1 CA3073648 A1 CA 3073648A1
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- bone
- acid sphingomyelinase
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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
- 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
- A61P19/00—Drugs for skeletal disorders
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P19/00—Drugs for skeletal disorders
- A61P19/08—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P19/00—Drugs for skeletal disorders
- A61P19/08—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
- A61P19/10—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease for osteoporosis
-
- 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
-
- 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)
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Physical Education & Sports Medicine (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
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- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Rheumatology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Genetics & Genomics (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Diabetes (AREA)
- Epidemiology (AREA)
- Hematology (AREA)
- Obesity (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Immunology (AREA)
- Gastroenterology & Hepatology (AREA)
- Microbiology (AREA)
- Biotechnology (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Enzymes And Modification Thereof (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762549732P | 2017-08-24 | 2017-08-24 | |
| US62/549,732 | 2017-08-24 | ||
| EP17306720 | 2017-12-07 | ||
| EP17306720.8 | 2017-12-07 | ||
| PCT/IB2018/056346 WO2019038685A2 (en) | 2017-08-24 | 2018-08-22 | TREATMENT OF ABNORMAL BONE CONDITIONS IN PATIENTS WITH ACIDIC SPHINGOMYELASE DISEASE |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CA3073648A1 true CA3073648A1 (en) | 2019-02-28 |
Family
ID=63556374
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA3073648A Pending CA3073648A1 (en) | 2017-08-24 | 2018-08-22 | Treatment of abnormal bone conditions in acid sphingomyelinase deficiency patients |
Country Status (17)
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| KR102094253B1 (ko) * | 2009-08-28 | 2020-03-31 | 이칸 스쿨 오브 메디슨 엣 마운트 시나이 | 산성 스핑고미엘리나제 결핍을 치료하기 위한 투여량 단계적 증가 효소 대체 요법 |
| UY38238A (es) * | 2018-05-25 | 2019-12-31 | Genzyme Corp | Composiciones farmacéuticas para el tratamiento de la deficiencia de esfingomielinasa ácida |
| JP7568648B2 (ja) * | 2019-05-31 | 2024-10-16 | ジェンザイム・コーポレーション | 二次元ｌｃ－ｍｓ／ｍｓシステム |
| CN112773779B (zh) * | 2021-02-04 | 2022-08-16 | 上海交通大学医学院附属新华医院 | 氨溴索、千金藤和汉防已在治疗酸性鞘磷脂酶缺乏症中的应用 |
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| US5773278A (en) | 1991-05-03 | 1998-06-30 | Mount Sinai Medical Center | Acid sphingomyelinase gene |
| CN1302897A (zh) | 1999-10-28 | 2001-07-11 | 上海博容基因开发有限公司 | 一种新的多肽——人酸性鞘磷酯酶相似的磷酸二酯酶21和编码这种多肽的多核苷酸 |
| WO2006130769A2 (en) * | 2005-06-02 | 2006-12-07 | Tercica, Inc. | Methods for treatment of growth disorders |
| KR102094253B1 (ko) * | 2009-08-28 | 2020-03-31 | 이칸 스쿨 오브 메디슨 엣 마운트 시나이 | 산성 스핑고미엘리나제 결핍을 치료하기 위한 투여량 단계적 증가 효소 대체 요법 |
| HK1207005A1 (en) | 2012-03-02 | 2016-01-22 | 夏尔人类遗传性治疗公司 | Compositions and methods for treating type iii gaucher disease |
| WO2014093354A1 (en) | 2012-12-12 | 2014-06-19 | Teva Pharmaceutical Industries Ltd. | Fusion of human growth hormone and albumin, formulation and uses thereof |
| PL3004896T3 (pl) | 2013-06-07 | 2020-04-30 | Genzyme Corporation | Marker zaburzeń kwaśnej spfingomielinazy i jego zastosowania |
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