HRP20210398T1 - Režimi doziranja za liječenje pompeove bolesti - Google Patents
Režimi doziranja za liječenje pompeove bolesti Download PDFInfo
- Publication number
- HRP20210398T1 HRP20210398T1 HRP20210398TT HRP20210398T HRP20210398T1 HR P20210398 T1 HRP20210398 T1 HR P20210398T1 HR P20210398T T HRP20210398T T HR P20210398TT HR P20210398 T HRP20210398 T HR P20210398T HR P20210398 T1 HRP20210398 T1 HR P20210398T1
- Authority
- HR
- Croatia
- Prior art keywords
- dnj
- pharmaceutically acceptable
- acceptable salt
- replacement therapy
- human recombinant
- Prior art date
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)
- 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)
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 |
|---|---|
| HRP20210398T1 true HRP20210398T1 (hr) | 2021-05-28 |
Family
ID=49514881
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| HRP20210398TT HRP20210398T1 (hr) | 2012-05-03 | 2013-05-02 | Režimi doziranja za liječenje 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)
| 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)
| 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 |
-
2013
- 2013-05-02 EP EP13785028.5A patent/EP2844279B1/en active Active
- 2013-05-02 EP EP24161179.7A patent/EP4397364A3/en active Pending
- 2013-05-02 JP JP2015510446A patent/JP6381521B2/ja active Active
- 2013-05-02 RS RS20210295A patent/RS61598B1/sr unknown
- 2013-05-02 SM SM20210136T patent/SMT202100136T1/it unknown
- 2013-05-02 ES ES20207542T patent/ES2980828T3/es active Active
- 2013-05-02 PL PL13785028T patent/PL2844279T3/pl unknown
- 2013-05-02 HU HUE13785028A patent/HUE053565T2/hu unknown
- 2013-05-02 WO PCT/US2013/039215 patent/WO2013166249A1/en not_active Ceased
- 2013-05-02 DK DK13785028.5T patent/DK2844279T3/da active
- 2013-05-02 EP EP20207542.0A patent/EP3871688B1/en active Active
- 2013-05-02 LT LTEP13785028.5T patent/LT2844279T/lt unknown
- 2013-05-02 ES ES13785028T patent/ES2859761T3/es active Active
- 2013-05-02 PT PT137850285T patent/PT2844279T/pt unknown
- 2013-05-02 US US14/398,210 patent/US20150086530A1/en active Pending
- 2013-05-02 HR HRP20210398TT patent/HRP20210398T1/hr unknown
- 2013-05-02 SI SI201331858T patent/SI2844279T1/sl unknown
-
2018
- 2018-01-19 JP JP2018007672A patent/JP6612370B2/ja active Active
-
2019
- 2019-10-29 JP JP2019196583A patent/JP6876111B2/ja active Active
-
2021
- 2021-01-27 JP JP2021010931A patent/JP7046241B2/ja active Active
- 2021-03-09 CY CY20211100202T patent/CY1124135T1/el unknown
-
2022
- 2022-03-22 JP JP2022044812A patent/JP2022101544A/ja not_active Withdrawn
-
2024
- 2024-03-07 JP JP2024034587A patent/JP2024081667A/ja active Pending
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 |
| 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 |
| RS61598B1 (sr) | 2021-04-29 |
| 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 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| CY1124135T1 (el) | Δοσολογικα σχηματα για την αγωγη της νοσου ρομρε | |
| JP2015519329A5 (enExample) | ||
| EA201270221A1 (ru) | Лечение болезни крона с применением лаквинимода | |
| HRP20191957T1 (hr) | Policiklični-karbamoilpiridonski spojevi i njihova farmaceutska upotreba | |
| MX2012000275A (es) | Antagonista de bomba de acido para el tratamiento de enfermedades involucradas en la motilidad gastrointestinal anormal. | |
| HRP20251310T1 (hr) | Enzimsko-nadomjesna terapija s povezivanjem doze za liječenje deficita kisele sfingomijelinaze | |
| HRP20191162T1 (hr) | Postupci liječenja ankilozantnog spondilitisa uporabom anti-il-17 protutijela | |
| HRP20161038T1 (hr) | Genz 112638 za liječenje gaucherove ili fabrijeve bolesti u kombiniranoj terapiji | |
| BR112015029512A8 (pt) | derivados de pirazolopirrolidina, seus usos, e composição e combinação farmacêuticas | |
| BR112015029491A2 (pt) | derivados de imidazopirrolidinona e seu uso no tratamento de doença | |
| BRPI0508461B8 (pt) | diaminopirimidinas, seus usos, e composição farmacêutica | |
| BR112012020236A2 (pt) | uso de um composto com atividade antagonística de ep4 ou um sal farmaceuticamente aceitável do mesmo, uso de um composto da fórmula (i), (ii), (iii), (iv), (va) ou (vb), ou um sal farmaceuticamente aceitável do mesmo, composição farmacêutica e método para o tratamento de doenças mediadas por il-23 em um sujeito animal, incluindo um sujeito mamífero | |
| JP2014528901A5 (enExample) | ||
| HRP20241454T1 (hr) | Migalastat za upotrebu u postupcima liječenja fabrijeve bolesti kod pacijenata koji imaju mutaciju u gla genu | |
| UA107578C2 (uk) | Комбінована терапія при лікуванні діабету | |
| AR082150A1 (es) | N-etil-n-fenil-1,2-dihidro-4-hidroxi-5-cloro-1-metil-2-oxoquinolin-3-carboxamida deuterada, sales y usos de la misma, metodo de tratamiento, mezcla compuestos, composicion farmaceutica, proceso de preparacion, compuesto enriquecido con deuterio | |
| BR112014003704A2 (pt) | moduladores ror gama | |
| JP2014513722A5 (enExample) | ||
| BR112014007357A2 (pt) | métodos de monitoramento terapêutico de fármacos de varredura de nitrogênio | |
| HRP20221366T1 (hr) | Postupci poboljšanja i/ili stabilizacije srčane funkcije kod bolesnika s fabrijevom bolešću | |
| HRP20180468T1 (hr) | Novi derivat analoga inzulina | |
| HRP20240025T1 (hr) | Liječenje bolesnika s klasičnom fabrijevom bolešću migalastatom | |
| AR080096A1 (es) | Tratamiento combinado de cancer de pancreas con gemcitabina y masitinib | |
| MX341660B (es) | Agonista del receptor de ghrelina para el tratamiento de caquexia. | |
| HRP20192246T1 (hr) | Marker za poremećaje kisele sfingomijelinaze i njegova uporaba |