CN111344003B - 酸性鞘磷脂酶缺乏症患者中异常骨病症的治疗 - Google Patents

酸性鞘磷脂酶缺乏症患者中异常骨病症的治疗 Download PDF

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Publication number
CN111344003B
CN111344003B CN201880067498.XA CN201880067498A CN111344003B CN 111344003 B CN111344003 B CN 111344003B CN 201880067498 A CN201880067498 A CN 201880067498A CN 111344003 B CN111344003 B CN 111344003B
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patient
leu
pro
rhasm
months
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Chinese (zh)
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CN111344003A (zh
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A·C·斯基特-普加
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Genzyme Corp
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Sanofi Aventis France
Genzyme Corp
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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)
  • Animal Behavior & Ethology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • 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)
CN201880067498.XA 2017-08-24 2018-08-22 酸性鞘磷脂酶缺乏症患者中异常骨病症的治疗 Active CN111344003B (zh)

Priority Applications (1)

Application Number Priority Date Filing Date Title
CN202410508209.2A CN118453843A (zh) 2017-08-24 2018-08-22 酸性鞘磷脂酶缺乏症患者中异常骨病症的治疗

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201762549732P 2017-08-24 2017-08-24
US62/549,732 2017-08-24
EP17306720.8 2017-12-07
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

Related Child Applications (1)

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CN202410508209.2A Division CN118453843A (zh) 2017-08-24 2018-08-22 酸性鞘磷脂酶缺乏症患者中异常骨病症的治疗

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CN111344003A CN111344003A (zh) 2020-06-26
CN111344003B true CN111344003B (zh) 2024-05-07

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CN201880067498.XA Active CN111344003B (zh) 2017-08-24 2018-08-22 酸性鞘磷脂酶缺乏症患者中异常骨病症的治疗
CN202410508209.2A Pending CN118453843A (zh) 2017-08-24 2018-08-22 酸性鞘磷脂酶缺乏症患者中异常骨病症的治疗

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US (2) US11898175B2 (enExample)
EP (1) EP3672622B1 (enExample)
JP (3) JP7216075B2 (enExample)
KR (2) KR102769578B1 (enExample)
CN (2) CN111344003B (enExample)
AU (2) AU2018319565B2 (enExample)
BR (1) BR112020003541A2 (enExample)
CA (1) CA3073648A1 (enExample)
CO (1) CO2020001801A2 (enExample)
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IL (1) IL272757B2 (enExample)
MX (2) MX2020002106A (enExample)
NZ (1) NZ762860A (enExample)
PL (1) PL3672622T3 (enExample)
SG (1) SG11202001544VA (enExample)
TW (1) TWI791040B (enExample)
WO (1) WO2019038685A2 (enExample)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX392330B (es) * 2009-08-28 2025-03-11 Icahn School Med Mount Sinai Uso farmacéutico de una esfingomielinasa ácida (asm) con aumento escalonado de la dosis para tratar la deficiencia de esfingomielinasa ácida.
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

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
CN102596232A (zh) * 2009-08-28 2012-07-18 西奈山医学院 用于治疗酸性鞘磷脂酶缺乏的剂量递增酶替代疗法
CN105474017A (zh) * 2013-06-07 2016-04-06 建新公司 酸性鞘磷脂酶病症的标志物及其用途

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5773278A (en) 1991-05-03 1998-06-30 Mount Sinai Medical Center Acid sphingomyelinase gene
EP1901770B1 (en) * 2005-06-02 2014-12-24 Ipsen Biopharmaceuticals, Inc. Gh and igf-1 for treatment of growth disorders
CA2865614A1 (en) 2012-03-02 2013-09-06 Shire Human Genetic Therapies, Inc. Compositions and methods for treating type iii gaucher disease
HK1216007A1 (zh) 2012-12-12 2016-10-07 Teva Pharmaceutical Industries Ltd. 人体生长激素和白蛋白的融合、制剂及其用途

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
CN102596232A (zh) * 2009-08-28 2012-07-18 西奈山医学院 用于治疗酸性鞘磷脂酶缺乏的剂量递增酶替代疗法
CN105999238A (zh) * 2009-08-28 2016-10-12 西奈山医学院 用于治疗酸性鞘磷脂酶缺乏的剂量递增酶替代疗法
CN106047835A (zh) * 2009-08-28 2016-10-26 西奈山医学院 用于治疗酸性鞘磷脂酶缺乏的剂量递增酶替代疗法
CN105474017A (zh) * 2013-06-07 2016-04-06 建新公司 酸性鞘磷脂酶病症的标志物及其用途

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
奥利普酶α治疗成年人酸性鞘磷脂酶缺乏症开始Ⅱ/Ⅲ期临床试验;黄世杰;;国际药学研究杂志;第43卷(第05期);第1007页 *

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AU2025203384A1 (en) 2025-05-29
US20210139868A1 (en) 2021-05-13
JP7431356B2 (ja) 2024-02-14
EP3672622B1 (en) 2023-11-08
CN111344003A (zh) 2020-06-26
NZ762860A (en) 2025-09-26
JP2024050732A (ja) 2024-04-10
CO2020001801A2 (es) 2020-04-01
TW201919689A (zh) 2019-06-01
KR102769578B1 (ko) 2025-02-20
KR20200044064A (ko) 2020-04-28
IL272757A (en) 2020-04-30
AU2018319565B2 (en) 2025-02-20
SG11202001544VA (en) 2020-03-30
MX2023010908A (es) 2023-09-27
WO2019038685A2 (en) 2019-02-28
ES2970423T3 (es) 2024-05-28
IL272757B1 (en) 2024-03-01
JP2023052459A (ja) 2023-04-11
CA3073648A1 (en) 2019-02-28
EP3672622A2 (en) 2020-07-01
AU2018319565A1 (en) 2020-04-09
MX2020002106A (es) 2020-07-14
JP2020531527A (ja) 2020-11-05
RU2020111649A (ru) 2021-09-24
US20240218338A1 (en) 2024-07-04
BR112020003541A2 (pt) 2020-09-01
KR20250025051A (ko) 2025-02-20
PL3672622T3 (pl) 2024-04-02
WO2019038685A3 (en) 2019-05-02
JP7216075B2 (ja) 2023-01-31
TWI791040B (zh) 2023-02-01
US11898175B2 (en) 2024-02-13
IL272757B2 (en) 2024-07-01
CN118453843A (zh) 2024-08-09

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