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 PDF

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Publication number
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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Prior art keywords
patient
rhasm
doses
bone
dose
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ES18768948T
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English (en)
Spanish (es)
Inventor
Ana Cristina Scheidt-Puga
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Genzyme Corp
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Genzyme Corp
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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • A61K38/465Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00Drugs for skeletal disorders
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00Drugs for skeletal disorders
    • A61P19/08Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00Drugs for skeletal disorders
    • A61P19/08Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
    • A61P19/10Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease for osteoporosis
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y301/00Hydrolases acting on ester bonds (3.1)
    • C12Y301/04Phosphoric 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)
ES18768948T 2017-08-24 2018-08-22 Esfingomielinasa ácida humana recombinante para su uso en el tratamiento de afecciones óseas anormales en pacientes con deficiencia de esfingomielinasa ácida Active ES2970423T3 (es)

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

Family

ID=63556374

Family Applications (1)

Application Number Title Priority Date Filing Date
ES18768948T Active ES2970423T3 (es) 2017-08-24 2018-08-22 Esfingomielinasa ácida humana recombinante para su uso en el tratamiento de afecciones óseas anormales en pacientes con deficiencia de esfingomielinasa ácida

Country Status (17)

Country Link
US (2) US11898175B2 (enExample)
EP (1) EP3672622B1 (enExample)
JP (3) JP7216075B2 (enExample)
KR (2) KR20250025051A (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)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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

Family Cites Families (7)

* 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
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

Also Published As

Publication number Publication date
MX2023010908A (es) 2023-09-27
AU2018319565A1 (en) 2020-04-09
KR20200044064A (ko) 2020-04-28
JP7216075B2 (ja) 2023-01-31
PL3672622T3 (pl) 2024-04-02
KR102769578B1 (ko) 2025-02-20
IL272757A (en) 2020-04-30
IL272757B1 (en) 2024-03-01
TWI791040B (zh) 2023-02-01
SG11202001544VA (en) 2020-03-30
JP2023052459A (ja) 2023-04-11
BR112020003541A2 (pt) 2020-09-01
TW201919689A (zh) 2019-06-01
AU2018319565B2 (en) 2025-02-20
CN118453843A (zh) 2024-08-09
EP3672622B1 (en) 2023-11-08
WO2019038685A2 (en) 2019-02-28
CN111344003B (zh) 2024-05-07
CA3073648A1 (en) 2019-02-28
JP2024050732A (ja) 2024-04-10
JP7431356B2 (ja) 2024-02-14
KR20250025051A (ko) 2025-02-20
MX2020002106A (es) 2020-07-14
EP3672622A2 (en) 2020-07-01
IL272757B2 (en) 2024-07-01
WO2019038685A3 (en) 2019-05-02
US20210139868A1 (en) 2021-05-13
JP2020531527A (ja) 2020-11-05
CO2020001801A2 (es) 2020-04-01
US11898175B2 (en) 2024-02-13
NZ762860A (en) 2025-09-26
CN111344003A (zh) 2020-06-26
RU2020111649A (ru) 2021-09-24
AU2025203384A1 (en) 2025-05-29
US20240218338A1 (en) 2024-07-04

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