JP6381521B2 - ポンペ病の処置のための投与計画 - Google Patents

ポンペ病の処置のための投与計画 Download PDF

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JP6381521B2
JP6381521B2 JP2015510446A JP2015510446A JP6381521B2 JP 6381521 B2 JP6381521 B2 JP 6381521B2 JP 2015510446 A JP2015510446 A JP 2015510446A JP 2015510446 A JP2015510446 A JP 2015510446A JP 6381521 B2 JP6381521 B2 JP 6381521B2
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deoxynojirimycin
administration
enzyme replacement
acid
glucosidase enzyme
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JP2015519329A5 (https=
JP2015519329A (ja
Inventor
ダグラス スチュアート グリーン，
ダグラス スチュアート グリーン，
ケネス ジョセフ バレンツァノ，
ケネス ジョセフ バレンツァノ，
Original Assignee
アミカス セラピューティックス インコーポレイテッド
アミカス セラピューティックス インコーポレイテッド
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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)
    • A61K38/47—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)

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  • 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)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Organic Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Epidemiology (AREA)
  • Diabetes (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Pulmonology (AREA)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Cardiology (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)
  • Hydrogenated Pyridines (AREA)
JP2015510446A 2012-05-03 2013-05-02 ポンペ病の処置のための投与計画 Active JP6381521B2 (ja)

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US201261642311P 2012-05-03 2012-05-03
US61/642,311 2012-05-03
US201261664011P 2012-06-25 2012-06-25
US61/664,011 2012-06-25
US201261697179P 2012-09-05 2012-09-05
US61/697,179 2012-09-05
US201361749234P 2013-01-04 2013-01-04
US201361749132P 2013-01-04 2013-01-04
US61/749,132 2013-01-04
US61/749,234 2013-01-04
PCT/US2013/039215 WO2013166249A1 (en) 2012-05-03 2013-05-02 Dosing regimens for the treatment of pompe disease

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2018007672A Division JP6612370B2 (ja) 2012-05-03 2018-01-19 ポンペ病の処置のための投与計画

Publications (3)

Publication Number Publication Date
JP2015519329A JP2015519329A (ja) 2015-07-09
JP2015519329A5 JP2015519329A5 (https=) 2018-03-08
JP6381521B2 true JP6381521B2 (ja) 2018-08-29

Family

ID=49514881

Family Applications (6)

Application Number Title Priority Date Filing Date
JP2015510446A Active JP6381521B2 (ja) 2012-05-03 2013-05-02 ポンペ病の処置のための投与計画
JP2018007672A Active JP6612370B2 (ja) 2012-05-03 2018-01-19 ポンペ病の処置のための投与計画
JP2019196583A Active JP6876111B2 (ja) 2012-05-03 2019-10-29 ポンペ病の処置のための投与計画
JP2021010931A Active JP7046241B2 (ja) 2012-05-03 2021-01-27 ポンペ病の処置のための投与計画
JP2022044812A Withdrawn JP2022101544A (ja) 2012-05-03 2022-03-22 ポンペ病の処置のための投与計画
JP2024034587A Pending JP2024081667A (ja) 2012-05-03 2024-03-07 ポンペ病の処置のための投与計画

Family Applications After (5)

Application Number Title Priority Date Filing Date
JP2018007672A Active JP6612370B2 (ja) 2012-05-03 2018-01-19 ポンペ病の処置のための投与計画
JP2019196583A Active JP6876111B2 (ja) 2012-05-03 2019-10-29 ポンペ病の処置のための投与計画
JP2021010931A Active JP7046241B2 (ja) 2012-05-03 2021-01-27 ポンペ病の処置のための投与計画
JP2022044812A Withdrawn JP2022101544A (ja) 2012-05-03 2022-03-22 ポンペ病の処置のための投与計画
JP2024034587A Pending JP2024081667A (ja) 2012-05-03 2024-03-07 ポンペ病の処置のための投与計画

Country Status (15)

Country Link
US (1) US20150086530A1 (https=)
EP (3) EP3871688B1 (https=)
JP (6) JP6381521B2 (https=)
CY (1) CY1124135T1 (https=)
DK (1) DK2844279T3 (https=)
ES (2) ES2859761T3 (https=)
HR (1) HRP20210398T1 (https=)
HU (1) HUE053565T2 (https=)
LT (1) LT2844279T (https=)
PL (1) PL2844279T3 (https=)
PT (1) PT2844279T (https=)
RS (1) RS61598B1 (https=)
SI (1) SI2844279T1 (https=)
SM (1) SMT202100136T1 (https=)
WO (1) WO2013166249A1 (https=)

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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
US8753674B2 (en) 2011-06-03 2014-06-17 Signpath Pharma Inc. Liposomal mitigation of drug-induced long QT syndrome and potassium delayed-rectifier current
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
US12004868B2 (en) 2011-06-03 2024-06-11 Signpath Pharma Inc. Liposomal mitigation of drug-induced inhibition of the cardiac IKr channel
US10532045B2 (en) 2013-12-18 2020-01-14 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
SMT202100136T1 (it) * 2012-05-03 2021-05-07 Amicus Therapeutics Inc Regimi posologici per il trattamento della malattia di pompe
US9675627B2 (en) * 2014-04-14 2017-06-13 Amicus Therapeutics, Inc. Dosing regimens for treating and/or preventing cerebral amyloidoses
AU2015269699B2 (en) 2014-06-03 2020-08-13 Avanti Polar Lipids, Inc. Protective effect of DMPC, DMPG, DMPC/DMPG, EGPG, LysoPG and LysoPC against drugs that cause channelopathies
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
TW202417620A (zh) * 2014-09-30 2024-05-01 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
KR20250007693A (ko) 2015-12-30 2025-01-14 아미쿠스 세라퓨틱스, 인코포레이티드 폼페병 치료용의 강화된 산 알파-글루코시다제
HRP20231465T1 (hr) 2015-12-30 2024-03-01 Amicus Therapeutics, Inc. Poboljšana kisela alfa-glukozidaza za liječenje pompeove bolesti
CA3019354A1 (en) 2016-03-30 2017-10-05 Amicus Therapeutics, Inc. Method for selection of high m6p recombinant proteins
AU2017239641A1 (en) 2016-03-30 2018-10-18 Amicus Therapeutics, Inc. Formulations comprising recombinant acid alpha-glucosidase
AU2017257496B2 (en) 2016-04-27 2020-05-07 Signpath Pharma, Inc. Prevention of drug-induced atrio-ventricular block
US11944628B2 (en) 2017-05-12 2024-04-02 Duke University Methods for the use of low-dose immune modulators transiently for treating patients undergoing protein replacement therapy
SMT202300187T1 (it) 2017-05-15 2023-07-20 Amicus Therapeutics Inc Alfa-glucosidasi acida umana ricombinante
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

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US6534300B1 (en) 1999-09-14 2003-03-18 Genzyme Glycobiology Research Institute, Inc. Methods for producing highly phosphorylated lysosomal hydrolases
AU2001269923A1 (en) 2000-06-19 2002-01-02 Genzyme Corporation Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases
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WO2002087510A2 (en) 2001-04-30 2002-11-07 Symbiontics, Inc. Subcellular targeting of therapeutic proteins
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HUE036449T2 (hu) 2003-06-24 2018-07-30 Genzyme Corp Új béta-aktin és RPS21 promóterek és alkalmazásaik
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SMT202100136T1 (it) * 2012-05-03 2021-05-07 Amicus Therapeutics Inc Regimi posologici per il trattamento della malattia di pompe

Also Published As

Publication number Publication date
HRP20210398T1 (hr) 2021-05-28
JP2022101544A (ja) 2022-07-06
PT2844279T (pt) 2021-03-11
JP6612370B2 (ja) 2019-11-27
JP2018109003A (ja) 2018-07-12
LT2844279T (lt) 2021-03-25
JP2020045346A (ja) 2020-03-26
SMT202100136T1 (it) 2021-05-07
ES2859761T3 (es) 2021-10-04
EP2844279A1 (en) 2015-03-11
JP2024081667A (ja) 2024-06-18
ES2980828T3 (es) 2024-10-03
US20150086530A1 (en) 2015-03-26
JP6876111B2 (ja) 2021-05-26
EP2844279B1 (en) 2020-12-09
EP2844279A4 (en) 2015-12-16
SI2844279T1 (sl) 2021-04-30
CY1124135T1 (el) 2022-05-27
PL2844279T3 (pl) 2021-08-16
EP3871688B1 (en) 2024-03-06
EP3871688A1 (en) 2021-09-01
JP2015519329A (ja) 2015-07-09
WO2013166249A1 (en) 2013-11-07
HUE053565T2 (hu) 2021-07-28
DK2844279T3 (da) 2021-03-15
EP4397364A3 (en) 2024-09-04
RS61598B1 (sr) 2021-04-29
EP4397364A2 (en) 2024-07-10
JP2021088561A (ja) 2021-06-10
JP7046241B2 (ja) 2022-04-01

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