RS61598B1 - Režimi doziranja za tretman pompeove bolesti - Google Patents

Režimi doziranja za tretman pompeove bolesti

Info

Publication number
RS61598B1
RS61598B1 RS20210295A RSP20210295A RS61598B1 RS 61598 B1 RS61598 B1 RS 61598B1 RS 20210295 A RS20210295 A RS 20210295A RS P20210295 A RSP20210295 A RS P20210295A RS 61598 B1 RS61598 B1 RS 61598B1
Authority
RS
Serbia
Prior art keywords
dnj
rhgaa
pharmaceutically acceptable
acceptable salt
glucosidase
Prior art date
Application number
RS20210295A
Other languages
English (en)
Serbian (sr)
Inventor
Douglas Stuart Greene
Kenneth Joseph Valenzano
Original Assignee
Amicus Therapeutics Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Amicus Therapeutics Inc filed Critical Amicus Therapeutics Inc
Publication of RS61598B1 publication Critical patent/RS61598B1/sr

Links

Classifications

    • 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)
    • A61K38/47Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/44Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/445Non condensed piperidines, e.g. piperocaine
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00Drugs for disorders of the respiratory system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • A61P3/08Drugs for disorders of the metabolism for glucose homeostasis
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y302/00Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/0102Alpha-glucosidase (3.2.1.20)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Chemical & Material Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Epidemiology (AREA)
  • Diabetes (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Endocrinology (AREA)
  • Cardiology (AREA)
  • Emergency Medicine (AREA)
  • Neurology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Pulmonology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Hydrogenated Pyridines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
RS20210295A 2012-05-03 2013-05-02 Režimi doziranja za tretman pompeove bolesti RS61598B1 (sr)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201261642311P 2012-05-03 2012-05-03
US201261664011P 2012-06-25 2012-06-25
US201261697179P 2012-09-05 2012-09-05
US201361749132P 2013-01-04 2013-01-04
US201361749234P 2013-01-04 2013-01-04
PCT/US2013/039215 WO2013166249A1 (en) 2012-05-03 2013-05-02 Dosing regimens for the treatment of pompe disease
EP13785028.5A EP2844279B1 (en) 2012-05-03 2013-05-02 Dosing regimens for the treatment of pompe disease

Publications (1)

Publication Number Publication Date
RS61598B1 true RS61598B1 (sr) 2021-04-29

Family

ID=49514881

Family Applications (1)

Application Number Title Priority Date Filing Date
RS20210295A RS61598B1 (sr) 2012-05-03 2013-05-02 Režimi doziranja za tretman pompeove bolesti

Country Status (15)

Country Link
US (1) US20150086530A1 (enExample)
EP (3) EP2844279B1 (enExample)
JP (6) JP6381521B2 (enExample)
CY (1) CY1124135T1 (enExample)
DK (1) DK2844279T3 (enExample)
ES (2) ES2980828T3 (enExample)
HR (1) HRP20210398T1 (enExample)
HU (1) HUE053565T2 (enExample)
LT (1) LT2844279T (enExample)
PL (1) PL2844279T3 (enExample)
PT (1) PT2844279T (enExample)
RS (1) RS61598B1 (enExample)
SI (1) SI2844279T1 (enExample)
SM (1) SMT202100136T1 (enExample)
WO (1) WO2013166249A1 (enExample)

Families Citing this family (20)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10349884B2 (en) 2011-06-03 2019-07-16 Sighpath Pharma Inc. Liposomal mitigation of drug-induced inhibition of the cardiac ikr channel
US10117881B2 (en) 2011-06-03 2018-11-06 Signpath Pharma, Inc. Protective effect of DMPC, DMPG, DMPC/DMPG, LYSOPG and LYSOPC against drugs that cause channelopathies
US10449193B2 (en) 2011-06-03 2019-10-22 Signpath Pharma Inc. Protective effect of DMPC, DMPG, DMPC/DMPG, lysoPG and lysoPC against drugs that cause channelopathies
US12004868B2 (en) 2011-06-03 2024-06-11 Signpath Pharma Inc. Liposomal mitigation of drug-induced inhibition of the cardiac IKr channel
US10238602B2 (en) 2011-06-03 2019-03-26 Signpath Pharma, Inc. Protective effect of DMPC, DMPG, DMPC/DMPG, LysoPG and LysoPC against drugs that cause channelopathies
WO2012167212A2 (en) 2011-06-03 2012-12-06 Signpath Pharma Inc. Liposomal mitigation of drug-induced long qt syndrome and potassium delayed-rectifier current
EP2844279B1 (en) * 2012-05-03 2020-12-09 Amicus Therapeutics, Inc. Dosing regimens for the treatment of pompe disease
WO2015095576A1 (en) * 2013-12-18 2015-06-25 Signpath Pharma, Inc. Liposomal mitigation of drug-induced inhibition of the cardiac ikr channel
US9675627B2 (en) * 2014-04-14 2017-06-13 Amicus Therapeutics, Inc. Dosing regimens for treating and/or preventing cerebral amyloidoses
EP3207931A3 (en) 2014-06-03 2017-12-20 Signpath Pharma Inc. Protective effect of dmpc, dmpg, dmpc/dmpg, egpg, lysopg and lysopc against drugs that cause channelopathies
TWI789758B (zh) * 2014-09-30 2023-01-11 美商阿米庫斯醫療股份有限公司 高效經修飾之酸性α-葡萄糖苷酶及其製造與使用方法
CN114540327B (zh) 2014-09-30 2026-03-24 阿米库斯治疗学公司 具有增强的碳水化合物的高强度酸性α-葡糖苷酶
KR102747683B1 (ko) 2015-12-30 2024-12-31 아미쿠스 세라퓨틱스, 인코포레이티드 폼페병 치료용의 강화된 산 알파-글루코시다제
RS64843B1 (sr) 2015-12-30 2023-12-29 Amicus Therapeutics Inc Augmentirana kisela alfa-glukozidaza za lečenje pompeove bolesti
JP7046003B2 (ja) 2016-03-30 2022-04-01 アミカス セラピューティックス インコーポレイテッド 高m6p組換えタンパク質の選択方法
SG11201808455VA (en) 2016-03-30 2018-10-30 Amicus Therapeutics Inc Formulations comprising recombinant acid alpha-glucosidase
KR102638618B1 (ko) 2016-04-27 2024-02-21 사인패스 파마 인코포레이티드 약물 유발된 방실 차단의 방지
WO2018209300A1 (en) * 2017-05-12 2018-11-15 Duke University Methods for the use of low-dose immune modulators transiently for treating patients undergoing protein replacement therapy
ES2950808T3 (es) 2017-05-15 2023-10-13 Amicus Therapeutics Inc Alfa-glucosidasa ácida humana recombinante
WO2022227287A1 (zh) * 2021-04-25 2022-11-03 中南大学 Dnj及其衍生物在制备预防和/或治疗肺动脉高压药物中的应用

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6642038B1 (en) 1999-09-14 2003-11-04 Genzyme Glycobiology Research Institute, Inc. GlcNAc phosphotransferase of the lysosomal targeting pathway
AU2001269923A1 (en) 2000-06-19 2002-01-02 Genzyme Corporation Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases
EP1974752B1 (en) 2001-04-30 2012-09-26 BioMarin Pharmaceutical Inc. Subcellular targeting of therapeutic proteins
US7560424B2 (en) 2001-04-30 2009-07-14 Zystor Therapeutics, Inc. Targeted therapeutic proteins
US20030072761A1 (en) 2001-10-16 2003-04-17 Lebowitz Jonathan Methods and compositions for targeting proteins across the blood brain barrier
WO2003086452A2 (en) 2002-04-05 2003-10-23 Genzyme Corporation Methods of enhancing lysosomal storage disease therapy
BRPI0411843B8 (pt) 2003-06-24 2021-05-25 Genzyme Corp ácido nucleico compreendendo um promotor de beta-actina, vetor e método de produzir uma proteína
AU2005211775B2 (en) 2004-02-06 2009-10-08 Biomarin Pharmaceutical Inc. Manufacture of highly phosphorylated lysosomal enzymes and uses thereof
CA2553955C (en) 2004-02-10 2012-08-28 Zystor Therapeutics, Inc. Acid alpha-glucosidase and fragments thereof
AU2006247065B8 (en) * 2005-05-17 2012-07-12 Amicus Therapeutics, Inc. A method for the treatment of Pompe disease using 1-deoxynojirimycin derivatives
WO2008134628A2 (en) * 2007-04-26 2008-11-06 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
WO2010056746A1 (en) * 2008-11-11 2010-05-20 Amicus Therapeutics, Inc. Therapy regimens, dosing regimens and stable medicaments for the treatment of pompe disease
EP2844279B1 (en) * 2012-05-03 2020-12-09 Amicus Therapeutics, Inc. Dosing regimens for the treatment of pompe disease

Also Published As

Publication number Publication date
JP2024081667A (ja) 2024-06-18
DK2844279T3 (da) 2021-03-15
EP2844279A4 (en) 2015-12-16
JP6876111B2 (ja) 2021-05-26
JP6612370B2 (ja) 2019-11-27
PL2844279T3 (pl) 2021-08-16
WO2013166249A1 (en) 2013-11-07
JP2020045346A (ja) 2020-03-26
JP7046241B2 (ja) 2022-04-01
HRP20210398T1 (hr) 2021-05-28
SI2844279T1 (sl) 2021-04-30
JP2022101544A (ja) 2022-07-06
CY1124135T1 (el) 2022-05-27
JP2015519329A (ja) 2015-07-09
ES2859761T3 (es) 2021-10-04
US20150086530A1 (en) 2015-03-26
PT2844279T (pt) 2021-03-11
SMT202100136T1 (it) 2021-05-07
EP2844279A1 (en) 2015-03-11
HUE053565T2 (hu) 2021-07-28
JP2018109003A (ja) 2018-07-12
ES2980828T3 (es) 2024-10-03
EP2844279B1 (en) 2020-12-09
JP2021088561A (ja) 2021-06-10
EP4397364A2 (en) 2024-07-10
EP3871688B1 (en) 2024-03-06
JP6381521B2 (ja) 2018-08-29
EP4397364A3 (en) 2024-09-04
EP3871688A1 (en) 2021-09-01
LT2844279T (lt) 2021-03-25

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