SI2844279T1 - Dozirni režimi za zdravljenje Pompejeve bolezni - Google Patents

Dozirni režimi za zdravljenje Pompejeve bolezni

Info

Publication number
SI2844279T1
SI2844279T1 SI201331858T SI201331858T SI2844279T1 SI 2844279 T1 SI2844279 T1 SI 2844279T1 SI 201331858 T SI201331858 T SI 201331858T SI 201331858 T SI201331858 T SI 201331858T SI 2844279 T1 SI2844279 T1 SI 2844279T1
Authority
SI
Slovenia
Prior art keywords
treatment
dosing regimens
pompe disease
pompe
disease
Prior art date
Application number
SI201331858T
Other languages
English (en)
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 SI2844279T1 publication Critical patent/SI2844279T1/sl

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)
  • Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Organic Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Diabetes (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Pulmonology (AREA)
  • Endocrinology (AREA)
  • Emergency Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
SI201331858T 2012-05-03 2013-05-02 Dozirni režimi za zdravljenje Pompejeve bolezni SI2844279T1 (sl)

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
EP13785028.5A EP2844279B1 (en) 2012-05-03 2013-05-02 Dosing regimens for the treatment of pompe disease
PCT/US2013/039215 WO2013166249A1 (en) 2012-05-03 2013-05-02 Dosing regimens for the treatment of pompe disease

Publications (1)

Publication Number Publication Date
SI2844279T1 true SI2844279T1 (sl) 2021-04-30

Family

ID=49514881

Family Applications (1)

Application Number Title Priority Date Filing Date
SI201331858T SI2844279T1 (sl) 2012-05-03 2013-05-02 Dozirni režimi za zdravljenje Pompejeve bolezni

Country Status (14)

Country Link
US (1) US20150086530A1 (sl)
EP (2) EP3871688B1 (sl)
JP (5) JP6381521B2 (sl)
CY (1) CY1124135T1 (sl)
DK (1) DK2844279T3 (sl)
ES (1) ES2859761T3 (sl)
HR (1) HRP20210398T1 (sl)
HU (1) HUE053565T2 (sl)
LT (1) LT2844279T (sl)
PL (1) PL2844279T3 (sl)
PT (1) PT2844279T (sl)
RS (1) RS61598B1 (sl)
SI (1) SI2844279T1 (sl)
WO (1) WO2013166249A1 (sl)

Families Citing this family (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
US8753674B2 (en) 2011-06-03 2014-06-17 Signpath Pharma Inc. Liposomal mitigation of drug-induced long QT syndrome and potassium delayed-rectifier current
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
US10349884B2 (en) 2011-06-03 2019-07-16 Sighpath Pharma Inc. Liposomal mitigation of drug-induced inhibition of the cardiac ikr channel
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
DK2844279T3 (da) * 2012-05-03 2021-03-15 Amicus Therapeutics Inc Doseringsprogrammer til behandling af pompes sygdom
CA2933204C (en) * 2013-12-18 2020-04-28 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
CA2950758C (en) 2014-06-03 2020-04-28 Signpath Pharma, Inc. Protective effect of dmpc, dmpg, dmpc/dmpg, egpg, lysopg and lysopc against drugs that cause channelopathies
ES2967620T3 (es) 2014-09-30 2024-05-03 Amicus Therapeutics Inc Alfa-glucosidasa ácida altamente potente con carbohidratos potenciados
TWI760296B (zh) * 2014-09-30 2022-04-11 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
BR112018013151A2 (pt) 2015-12-30 2018-12-18 Amicus Therapeutics Inc alfa-glicosidase ácida aumentada para o tratamento da doença de pompe
FI3957320T3 (fi) 2015-12-30 2023-11-23 Amicus Therapeutics Inc Vahvistettu hapan alfa-glukosidaasi pompen taudin hoitoon
US10227577B2 (en) 2016-03-30 2019-03-12 Amicus Therapeutics, Inc. Method for selection of high M6P recombinant proteins
US10512676B2 (en) 2016-03-30 2019-12-24 Amicus Therapeutics, Inc. Formulations comprising recombinant acid alpha-glucosidase
US11806401B2 (en) 2016-04-27 2023-11-07 Signpath Pharma, Inc. Prevention of drug-induced atrio-ventricular block
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
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
US6537785B1 (en) 1999-09-14 2003-03-25 Genzyme Glycobiology Research Institute, Inc. Methods of treating lysosomal storage diseases
AU2001269923A1 (en) 2000-06-19 2002-01-02 Genzyme Corporation Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases
US7560424B2 (en) 2001-04-30 2009-07-14 Zystor Therapeutics, Inc. Targeted therapeutic proteins
ES2300439T3 (es) 2001-04-30 2008-06-16 Zystor Therapeutics , Inc. Reconocimiento subcelular de proteinas terapeuticas.
US20030072761A1 (en) 2001-10-16 2003-04-17 Lebowitz Jonathan Methods and compositions for targeting proteins across the blood brain barrier
US7658916B2 (en) 2002-04-05 2010-02-09 Genzyme Corporation Methods of enhancing lysosomal storage disease therapy by modulation of cell surface receptor density
CN107058313A (zh) 2003-06-24 2017-08-18 建新公司 新的β‑肌动蛋白和RPS21启动子及其应用
WO2005077093A2 (en) 2004-02-06 2005-08-25 Biomarin Pharmaceutical Inc. Manufacture of highly phosphorylated lysosomal enzymes and uses thereof
US20050244400A1 (en) 2004-02-10 2005-11-03 Zystor Therapeutics, Inc. Acid alpha-glucosidase and fragments thereof
KR20080025373A (ko) 2005-05-17 2008-03-20 아미쿠스 세라퓨틱스, 인코포레이티드 1-데옥시노지리마이신 및 유도체를 이용한 폼페병의 치료방법
MX2009011473A (es) * 2007-04-26 2010-01-18 Amicus Therapeutics Inc Regimenes de dosificacion para el tratamiento de enfermedades por almacenamiento lisosomal que utilizan chaperonas farmacologicas.
US20100119502A1 (en) * 2008-11-11 2010-05-13 Amicus Therapeutics, Inc. Therapy regimens, dosing regimens and stable medicaments for the treatment of pompe disease
DK2844279T3 (da) * 2012-05-03 2021-03-15 Amicus Therapeutics Inc Doseringsprogrammer til behandling af pompes sygdom

Also Published As

Publication number Publication date
PT2844279T (pt) 2021-03-11
JP2015519329A (ja) 2015-07-09
JP7046241B2 (ja) 2022-04-01
EP2844279B1 (en) 2020-12-09
EP2844279A4 (en) 2015-12-16
JP2021088561A (ja) 2021-06-10
JP2018109003A (ja) 2018-07-12
JP2022101544A (ja) 2022-07-06
DK2844279T3 (da) 2021-03-15
HRP20210398T1 (hr) 2021-05-28
US20150086530A1 (en) 2015-03-26
JP6612370B2 (ja) 2019-11-27
LT2844279T (lt) 2021-03-25
PL2844279T3 (pl) 2021-08-16
JP2020045346A (ja) 2020-03-26
RS61598B1 (sr) 2021-04-29
JP6876111B2 (ja) 2021-05-26
EP3871688A1 (en) 2021-09-01
EP3871688B1 (en) 2024-03-06
JP6381521B2 (ja) 2018-08-29
HUE053565T2 (hu) 2021-07-28
EP2844279A1 (en) 2015-03-11
CY1124135T1 (el) 2022-05-27
WO2013166249A1 (en) 2013-11-07
ES2859761T3 (es) 2021-10-04

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