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 (enExample
JP2015519329A (ja
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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，
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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)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • General Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Epidemiology (AREA)
  • Diabetes (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Endocrinology (AREA)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Pulmonology (AREA)
  • Cardiology (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)
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 (enExample) 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 (enExample)
EP (3) EP3871688B1 (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)

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EP2714011B1 (en) 2011-06-03 2018-01-31 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
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
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
US12004868B2 (en) 2011-06-03 2024-06-11 Signpath Pharma Inc. Liposomal mitigation of drug-induced inhibition of the cardiac IKr channel
PT2844279T (pt) * 2012-05-03 2021-03-11 Amicus Therapeutics Inc Esquema posológico para o tratamento da doença de pompe
JP6895252B2 (ja) * 2013-12-18 2021-06-30 サインパス ファルマ, インク.Signpath Pharma, Inc. 心筋ｉｋｒチャネルの薬剤誘発性阻害のリポソームによる軽減
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
KR20250004926A (ko) 2014-09-30 2025-01-08 아미쿠스 세라퓨틱스, 인코포레이티드 개선된 탄수화물을 가지는 고도로 강력한 산성 알파-글루코시다제
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
KR102510941B1 (ko) 2015-12-30 2023-03-20 아미쿠스 세라퓨틱스, 인코포레이티드 폼페병 치료용의 강화된 산 알파-글루코시다제
CN108472340A (zh) 2015-12-30 2018-08-31 阿米库斯治疗学公司 用于治疗庞贝氏病的增强的酸性α-葡糖苷酶
WO2017173060A1 (en) 2016-03-30 2017-10-05 Amicus Therapeutics, Inc. Formulations comprising recombinant acid alpha-glucosidase
KR102790256B1 (ko) 2016-03-30 2025-04-04 아미쿠스 세라퓨틱스, 인코포레이티드 고 m6p 재조합 단백질의 선택 방법
AU2017257496B2 (en) 2016-04-27 2020-05-07 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
DK3624831T5 (da) 2017-05-15 2024-09-02 Amicus Therapeutics Inc Rekombinant human sur alfa-glucosidase
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
US20020095135A1 (en) 2000-06-19 2002-07-18 David Meeker 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
ATE384736T1 (de) 2001-04-30 2008-02-15 Zystor Therapeutics Inc Subzelluläres targeting von therapeutischen proteinen
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
EP3346010A1 (en) 2003-06-24 2018-07-11 Genzyme Corporation Novel beta-actin and rps21 promoters and uses thereof
US20080014188A1 (en) 2004-02-06 2008-01-17 Zankel Todd C Manufacture of Highly Phosphorylated Lysosomal Enzymes and Uses Thereof
JP4914224B2 (ja) 2004-02-10 2012-04-11 バイオマリン ファーマシューティカル インコーポレイテッド 酸性αグルコシダーゼおよびそのフラグメント
EP2932982B1 (en) * 2005-05-17 2018-10-03 Amicus Therapeutics, Inc. A method for the treatment of pompe disease using 1-deoxynojirimycin and derivatives
US9056101B2 (en) * 2007-04-26 2015-06-16 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
PT2844279T (pt) * 2012-05-03 2021-03-11 Amicus Therapeutics Inc Esquema posológico para o tratamento da doença de pompe

Also Published As

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

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