CY1124135T1 - Δοσολογικα σχηματα για την αγωγη της νοσου ρομρε - Google Patents

Δοσολογικα σχηματα για την αγωγη της νοσου ρομρε

Info

Publication number
CY1124135T1
CY1124135T1 CY20211100202T CY211100202T CY1124135T1 CY 1124135 T1 CY1124135 T1 CY 1124135T1 CY 20211100202 T CY20211100202 T CY 20211100202T CY 211100202 T CY211100202 T CY 211100202T CY 1124135 T1 CY1124135 T1 CY 1124135T1
Authority
CY
Cyprus
Prior art keywords
treatment
disease
romre
dosage forms
deoxynorrimycin
Prior art date
Application number
CY20211100202T
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 CY1124135T1 publication Critical patent/CY1124135T1/el

Links

Classifications

    • 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)
    • A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435—Heterocyclic 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
    • A61K31/44—Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/445—Non condensed piperidines, e.g. piperocaine
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • 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
    • 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
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/0102—Alpha-glucosidase (3.2.1.20)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Epidemiology (AREA)
  • Diabetes (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Cardiology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Pulmonology (AREA)
  • Endocrinology (AREA)
  • Emergency Medicine (AREA)
  • Heart & Thoracic Surgery (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)
  • Hydrogenated Pyridines (AREA)

Abstract

Το αντικείμενο που αποκαλύπτεται στο παρόν παρέχει ένα δοσολογικό σχήμα και ένα πρόγραμμα χορήγησης για τη χρήση της 1-δεοξυνοριριμυκίνης και της θεραπείας ενζυμικής υποκατάστασης (ERT) για την αγωγή της νόσου Pompe. To παρόν αποκαλυπτόμενο αντικείμενο παρέχει περαιτέρω ένα δοσολογικό σχήμα και ένα πρόγραμμα χορήγησης για τη χρήση της υδροχλωρικής 1-δεοξυνοριριμυκίνης και της αλγλυκοσιδάσης άλφα για την αγωγή της νόσου Pompe.
CY20211100202T 2012-05-03 2021-03-09 Δοσολογικα σχηματα για την αγωγη της νοσου ρομρε CY1124135T1 (el)

Applications Claiming Priority (6)

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

Publications (1)

Publication Number Publication Date
CY1124135T1 true CY1124135T1 (el) 2022-05-27

Family

ID=49514881

Family Applications (1)

Application Number Title Priority Date Filing Date
CY20211100202T CY1124135T1 (el) 2012-05-03 2021-03-09 Δοσολογικα σχηματα για την αγωγη της νοσου ρομρε

Country Status (14)

Country Link
US (1) US20150086530A1 (el)
EP (3) EP3871688B1 (el)
JP (6) JP6381521B2 (el)
CY (1) CY1124135T1 (el)
DK (1) DK2844279T3 (el)
ES (1) ES2859761T3 (el)
HR (1) HRP20210398T1 (el)
HU (1) HUE053565T2 (el)
LT (1) LT2844279T (el)
PL (1) PL2844279T3 (el)
PT (1) PT2844279T (el)
RS (1) RS61598B1 (el)
SI (1) SI2844279T1 (el)
WO (1) WO2013166249A1 (el)

Families Citing this family (19)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
US10349884B2 (en) 2011-06-03 2019-07-16 Sighpath Pharma Inc. Liposomal mitigation of drug-induced inhibition of the cardiac ikr channel
CA2836904C (en) 2011-06-03 2019-09-24 Signpath Pharma Inc. Liposomal mitigation of drug-induced long qt syndrome and potassium delayed-rectifier current
US12004868B2 (en) 2011-06-03 2024-06-11 Signpath 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
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
PL2844279T3 (pl) * 2012-05-03 2021-08-16 Amicus Therapeutics, Inc. Schematy dawkowania w leczeniu choroby pompego
EP3082768B1 (en) * 2013-12-18 2023-02-22 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
ES2942132T3 (es) 2014-06-03 2023-05-30 Signpath Pharma Inc Efecto protector de DMPC, DMPG, DMPC/DMPG, EGPG, LYSOPG y LYSOPC frente a fármacos que provocan canalopatías
CN114540327A (zh) 2014-09-30 2022-05-27 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
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
EP3397273B1 (en) 2015-12-30 2021-05-19 Amicus Therapeutics, Inc. Augmented acid alpha-glucosidase for the treatment of pompe disease
WO2017117407A1 (en) 2015-12-30 2017-07-06 Amicus Therapeutics, Inc. Augmented acid alpha-glucosidase for the treatment of pompe disease
SG11201808455VA (en) 2016-03-30 2018-10-30 Amicus Therapeutics Inc Formulations comprising recombinant acid alpha-glucosidase
CA3019354A1 (en) 2016-03-30 2017-10-05 Amicus Therapeutics, Inc. Method for selection of high m6p recombinant proteins
CA3038813C (en) 2016-04-27 2021-08-24 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
JP4641705B2 (ja) 2001-04-30 2011-03-02 ザイストール セラピューティクス， インコーポレイテッド 治療的タンパク質の亜細胞標的化
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
CN101942442B (zh) 2003-06-24 2013-10-09 建新公司 新的β-肌动蛋白和RPS21启动子及其应用
AU2005211775B2 (en) 2004-02-06 2009-10-08 Biomarin Pharmaceutical Inc. Manufacture of highly phosphorylated lysosomal enzymes and uses thereof
DE602005020745D1 (de) 2004-02-10 2010-06-02 Zystor Therapeutics Inc Saure alpha-glukosidase und fragmente davon
ES2705335T3 (es) * 2005-05-17 2019-03-22 Amicus Therapeutics Inc Un método para el tratamiento de la enfermedad de Pompe usando 1-desoxinojirimicina y derivados
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.
WO2010056746A1 (en) * 2008-11-11 2010-05-20 Amicus Therapeutics, Inc. Therapy regimens, dosing regimens and stable medicaments for the treatment of pompe disease
PL2844279T3 (pl) * 2012-05-03 2021-08-16 Amicus Therapeutics, Inc. Schematy dawkowania w leczeniu choroby pompego

Also Published As

Publication number Publication date
HUE053565T2 (hu) 2021-07-28
SI2844279T1 (sl) 2021-04-30
PT2844279T (pt) 2021-03-11
EP3871688B1 (en) 2024-03-06
EP2844279A1 (en) 2015-03-11
HRP20210398T1 (hr) 2021-05-28
JP2021088561A (ja) 2021-06-10
EP2844279B1 (en) 2020-12-09
JP2022101544A (ja) 2022-07-06
JP2015519329A (ja) 2015-07-09
EP3871688A1 (en) 2021-09-01
ES2859761T3 (es) 2021-10-04
DK2844279T3 (da) 2021-03-15
EP4397364A3 (en) 2024-09-04
WO2013166249A1 (en) 2013-11-07
PL2844279T3 (pl) 2021-08-16
EP4397364A2 (en) 2024-07-10
JP6612370B2 (ja) 2019-11-27
JP7046241B2 (ja) 2022-04-01
JP2018109003A (ja) 2018-07-12
JP2020045346A (ja) 2020-03-26
JP6381521B2 (ja) 2018-08-29
US20150086530A1 (en) 2015-03-26
EP2844279A4 (en) 2015-12-16
JP6876111B2 (ja) 2021-05-26
JP2024081667A (ja) 2024-06-18
LT2844279T (lt) 2021-03-25
RS61598B1 (sr) 2021-04-29

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