JP7216075B2 - 酸性スフィンゴミエリナーゼ欠損症患者における骨状態異常の処置 - Google Patents

酸性スフィンゴミエリナーゼ欠損症患者における骨状態異常の処置 Download PDF

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JP7216075B2
JP7216075B2 JP2020511325A JP2020511325A JP7216075B2 JP 7216075 B2 JP7216075 B2 JP 7216075B2 JP 2020511325 A JP2020511325 A JP 2020511325A JP 2020511325 A JP2020511325 A JP 2020511325A JP 7216075 B2 JP7216075 B2 JP 7216075B2
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patient
bone
acid sphingomyelinase
rhasm
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JP2020531527A (ja
JP2020531527A5 (enExample
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アナ・クリスティーナ・シャイト－プガ
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Sanofi SA
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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)
JP2020511325A 2017-08-24 2018-08-22 酸性スフィンゴミエリナーゼ欠損症患者における骨状態異常の処置 Active JP7216075B2 (ja)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2023005602A JP7431356B2 (ja) 2017-08-24 2023-01-18 酸性スフィンゴミエリナーゼ欠損症患者における骨状態異常の処置
JP2024012534A JP2024050732A (ja) 2017-08-24 2024-01-31 酸性スフィンゴミエリナーゼ欠損症患者における骨状態異常の処置

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

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JP2023005602A Division JP7431356B2 (ja) 2017-08-24 2023-01-18 酸性スフィンゴミエリナーゼ欠損症患者における骨状態異常の処置

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JP2020531527A JP2020531527A (ja) 2020-11-05
JP2020531527A5 JP2020531527A5 (enExample) 2021-09-30
JP7216075B2 true JP7216075B2 (ja) 2023-01-31

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JP2020511325A Active JP7216075B2 (ja) 2017-08-24 2018-08-22 酸性スフィンゴミエリナーゼ欠損症患者における骨状態異常の処置
JP2023005602A Active JP7431356B2 (ja) 2017-08-24 2023-01-18 酸性スフィンゴミエリナーゼ欠損症患者における骨状態異常の処置
JP2024012534A Pending JP2024050732A (ja) 2017-08-24 2024-01-31 酸性スフィンゴミエリナーゼ欠損症患者における骨状態異常の処置

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JP2023005602A Active JP7431356B2 (ja) 2017-08-24 2023-01-18 酸性スフィンゴミエリナーゼ欠損症患者における骨状態異常の処置
JP2024012534A Pending JP2024050732A (ja) 2017-08-24 2024-01-31 酸性スフィンゴミエリナーゼ欠損症患者における骨状態異常の処置

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US (2) US11898175B2 (enExample)
EP (1) EP3672622B1 (enExample)
JP (3) JP7216075B2 (enExample)
KR (2) KR102769578B1 (enExample)
CN (2) CN118453843A (enExample)
AU (2) AU2018319565B2 (enExample)
BR (1) BR112020003541A2 (enExample)
CA (1) CA3073648A1 (enExample)
CO (1) CO2020001801A2 (enExample)
ES (1) ES2970423T3 (enExample)
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)

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BR112012004377A2 (pt) * 2009-08-28 2017-12-12 Genzyme Corp terapia de reposição enzimática com progressão de dose para tratamento de deficiência de esfingomielinase ácida.
UY38238A (es) * 2018-05-25 2019-12-31 Genzyme Corp Composiciones farmacéuticas para el tratamiento de la deficiencia de esfingomielinasa ácida
HUE071194T2 (hu) * 2019-05-31 2025-08-28 Genzyme Corp Kétdimenziós LC-MS/MS rendszerek
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 (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2013502933A (ja) 2009-08-28 2013-01-31 マウント シナイ スクール オブ メディスン 酸性スフィンゴミエリナーゼ欠乏症に対する用量漸増酵素補充療法

Family Cites Families (6)

* 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
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
ES2532089T3 (es) * 2005-06-02 2015-03-24 Ipsen Biopharmaceuticals, Inc. GH e IGF-1 para el tratamiento de trastornos del crecimiento
KR102096752B1 (ko) 2012-03-02 2020-04-02 샤이어 휴먼 지네틱 테라피즈 인크. Ｉｉｉ형 고셔병을 치료하기 위한 조성물 및 방법
EA201591108A1 (ru) 2012-12-12 2015-11-30 Тева Фармасьютикал Индастриз Лтд. Слитый гормон роста человека и альбумин, составы и их применения
KR102228367B1 (ko) 2013-06-07 2021-03-16 젠자임 코포레이션 산 스핑고미엘리나제 장애에 대한 마커 및 이의 용도

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2013502933A (ja) 2009-08-28 2013-01-31 マウント シナイ スクール オブ メディスン 酸性スフィンゴミエリナーゼ欠乏症に対する用量漸増酵素補充療法

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
Disease manifestations and burden of illness in patients with acid sphingomyelinase deficiency (ASMD)，Orphanet J Rare Dis，2017年02月23日，Vol. 12, No. 1，Article number: 41，doi: 10.1186/s13023-017-0572-x
Successful within-patient dose escalation of olipudase alfa in acid sphingomyelinase deficiency，Molecular Genetics and Metabolism，2015年，Volume 116, Issues 12，Pages 88-97，https://doi.org/10.1016/j.ymgme.2015.05.013

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AU2018319565A1 (en) 2020-04-09
EP3672622A2 (en) 2020-07-01
KR20200044064A (ko) 2020-04-28
KR102769578B1 (ko) 2025-02-20
JP2024050732A (ja) 2024-04-10
US20210139868A1 (en) 2021-05-13
SG11202001544VA (en) 2020-03-30
US20240218338A1 (en) 2024-07-04
MX2020002106A (es) 2020-07-14
TW201919689A (zh) 2019-06-01
WO2019038685A2 (en) 2019-02-28
WO2019038685A3 (en) 2019-05-02
IL272757A (en) 2020-04-30
CN118453843A (zh) 2024-08-09
US11898175B2 (en) 2024-02-13
EP3672622B1 (en) 2023-11-08
PL3672622T3 (pl) 2024-04-02
MX2023010908A (es) 2023-09-27
JP2023052459A (ja) 2023-04-11
TWI791040B (zh) 2023-02-01
JP2020531527A (ja) 2020-11-05
BR112020003541A2 (pt) 2020-09-01
ES2970423T3 (es) 2024-05-28
IL272757B1 (en) 2024-03-01
IL272757B2 (en) 2024-07-01
AU2025203384A1 (en) 2025-05-29
AU2018319565B2 (en) 2025-02-20
NZ762860A (en) 2025-09-26
CN111344003B (zh) 2024-05-07
KR20250025051A (ko) 2025-02-20
CO2020001801A2 (es) 2020-04-01
RU2020111649A (ru) 2021-09-24
CN111344003A (zh) 2020-06-26
JP7431356B2 (ja) 2024-02-14
CA3073648A1 (en) 2019-02-28

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