ES2970423T3 - Esfingomielinasa ácida humana recombinante para su uso en el tratamiento de afecciones óseas anormales en pacientes con deficiencia de esfingomielinasa ácida - Google Patents
Esfingomielinasa ácida humana recombinante para su uso en el tratamiento de afecciones óseas anormales en pacientes con deficiencia de esfingomielinasa ácida Download PDFInfo
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- ES2970423T3 ES2970423T3 ES18768948T ES18768948T ES2970423T3 ES 2970423 T3 ES2970423 T3 ES 2970423T3 ES 18768948 T ES18768948 T ES 18768948T ES 18768948 T ES18768948 T ES 18768948T ES 2970423 T3 ES2970423 T3 ES 2970423T3
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Classifications
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- A—HUMAN NECESSITIES
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- 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
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- 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
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- 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
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- 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
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- 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
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- 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)
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- 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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- Bioinformatics & Cheminformatics (AREA)
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- Immunology (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Microbiology (AREA)
- Biomedical Technology (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 (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762549732P | 2017-08-24 | 2017-08-24 | |
| EP17306720 | 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 |
|---|---|
| ES2970423T3 true ES2970423T3 (es) | 2024-05-28 |
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| CA2773133A1 (en) * | 2009-08-28 | 2011-03-03 | Edward H. Schuchman | Dose escalation enzyme replacement therapy for treating acid sphingomyelinase deficiency |
| PL3672622T3 (pl) * | 2017-08-24 | 2024-04-02 | Genzyme Corporation | Rekombinowana ludzka kwaśna sfingomielinaza do zastosowania w leczeniu nieprawidłowych stanów kości u pacjentów z niedoborem kwaśnej sfingomielinazy |
| PY1939780A (es) * | 2018-05-25 | 2019-12-04 | 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 |
| TW202403043A (zh) * | 2022-03-18 | 2024-01-16 | 美商健臻公司 | 重組人類酸性鞘磷脂酶醫藥組合物及方法 |
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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 |
| US8158582B2 (en) * | 2005-06-02 | 2012-04-17 | Tercica, Inc. | Methods for treatment of insulin-like growth factor-1 deficiency |
| CA2773133A1 (en) * | 2009-08-28 | 2011-03-03 | Edward H. Schuchman | Dose escalation enzyme replacement therapy for treating acid sphingomyelinase deficiency |
| WO2013130963A1 (en) | 2012-03-02 | 2013-09-06 | Shire Human Genetic Therapies, Inc. | Compositions and methods for treating type iii gaucher disease |
| AR093908A1 (es) | 2012-12-12 | 2015-06-24 | Teva Pharma | Fusion de la hormona de crecimiento humana y albumina, formulacion y usos de la misma |
| MX370570B (es) * | 2013-06-07 | 2019-12-17 | Genzyme Corp | Marcador para trastornos de la esfingomielinasa acida y sus usos. |
| PL3672622T3 (pl) * | 2017-08-24 | 2024-04-02 | Genzyme Corporation | Rekombinowana ludzka kwaśna sfingomielinaza do zastosowania w leczeniu nieprawidłowych stanów kości u pacjentów z niedoborem kwaśnej sfingomielinazy |
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