DK2490533T3 - Hidtil ukendte sammensætninger til forebyggelse og/eller behandling af lysosomale ophobningssygdomme - Google Patents
Hidtil ukendte sammensætninger til forebyggelse og/eller behandling af lysosomale ophobningssygdomme Download PDFInfo
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- DK2490533T3 DK2490533T3 DK10825397.2T DK10825397T DK2490533T3 DK 2490533 T3 DK2490533 T3 DK 2490533T3 DK 10825397 T DK10825397 T DK 10825397T DK 2490533 T3 DK2490533 T3 DK 2490533T3
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- Prior art keywords
- compound
- hydrogen
- halogen
- diol
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- 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
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D211/00—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings
- C07D211/04—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom
- C07D211/06—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members
- C07D211/08—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members with hydrocarbon or substituted hydrocarbon radicals directly attached to ring carbon atoms
- C07D211/18—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members with hydrocarbon or substituted hydrocarbon radicals directly attached to ring carbon atoms with substituted hydrocarbon radicals attached to ring carbon atoms
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D211/00—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings
- C07D211/04—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom
- C07D211/06—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members
- C07D211/36—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members with hetero atoms or with carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals, directly attached to ring carbon atoms
- C07D211/40—Oxygen atoms
- C07D211/44—Oxygen atoms attached in position 4
- C07D211/46—Oxygen atoms attached in position 4 having a hydrogen atom as the second substituent in position 4
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- 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
- A61K31/13—Amines
- A61K31/15—Oximes (>C=N—O—); Hydrazines (>N—N<); Hydrazones (>N—N=) ; Imines (C—N=C)
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- 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
-
- 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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/20—Pills, tablets, discs, rods
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
- A61P1/04—Drugs for disorders of the alimentary tract or the digestive system for ulcers, gastritis or reflux esophagitis, e.g. antacids, inhibitors of acid secretion, mucosal protectants
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
- A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
- A61P1/18—Drugs for disorders of the alimentary tract or the digestive system for pancreatic disorders, e.g. pancreatic enzymes
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P17/00—Drugs for dermatological disorders
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P19/00—Drugs for skeletal disorders
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous 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
- A61P3/06—Antihyperlipidemics
-
- 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
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
- A61P7/06—Antianaemics
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
- A61P7/10—Antioedematous agents; Diuretics
-
- 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
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D211/00—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings
- C07D211/92—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with a hetero atom directly attached to the ring nitrogen atom
- C07D211/96—Sulfur atom
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- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Life Sciences & Earth Sciences (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- General Health & Medical Sciences (AREA)
- Animal Behavior & Ethology (AREA)
- Medicinal Chemistry (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Epidemiology (AREA)
- Hematology (AREA)
- Diabetes (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Immunology (AREA)
- Obesity (AREA)
- Neurology (AREA)
- Dermatology (AREA)
- Neurosurgery (AREA)
- Physical Education & Sports Medicine (AREA)
- Biomedical Technology (AREA)
- Heart & Thoracic Surgery (AREA)
- Cardiology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Hydrogenated Pyridines (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Claims (13)
1. Forbindelse med formlen II:
hvor: R1 er C (R2) (R3) (R4) ; R2 er hydrogen, -OH eller halogen; R3 er hydrogen, -OH, halogen eller -CH3; R4 er halogen, -CH3, phenyl, fluorphenyl, methylphenyl, cyclohexylmethyl, hvor R2 og R3 ikke begge kan være hydrogen, når R4 er halogen; R3 og R4 sammen med det carbonatom, hvortil de er bundet, kan udgøre en cycloalkylring, som eventuelt kan være substitueret med et eller flere halogenatomer; R6 er hydrogen eller phenylalkyl; Z er valgfri, og når Z er til stede, er det -(CH2)-, -C(=0)-, -S(=0)2NH-, -S(=0)2-, -S(=0)2-CH2-, C(=0)-NH-, -S(=0)2-NR9-, - C (=S) NH- eller -C(=0)CH2-; R9 er hydrogen eller CH3; R5 er hydrogen eller aminophenylalkyl; R7 er -OH eller halogen; og R8 er hydrogen, halogen eller -CH3, forudsat at R2 og R3 ikke begge kan være hydrogen, når R4 er halogen, og at når Z ikke er til stede, er R7 -OH, og R5, R6 og R8 er hydrogen; eller et farmaceutisk acceptabelt salt eller solvat deraf; til anvendelse til forebyggelse og/eller behandling af en lysosomal ophobningssygdom hos en patient, som har risiko for at udvikle eller er diagnosticeret med en sådan sygdom.
2. Forbindelse til anvendelse ifølge krav 1, hvor forbindelsen har formlen III:
hvor R1 er C (R2) (R3) (R4) ; R2 er hydrogen, -OH eller halogen; R3 er hydrogen, -OH, halogen eller -CH3; R4 er halogen, -CH3, phenyl, fluorphenyl, methylphenyl, cyclohexylmethyl, hvor R2 og R3 ikke begge kan være hydrogen, når R4 er halogen; R3 og R4 sammen med det carbonatom, hvortil de er bundet, kan udgøre en cycloalkylring, som eventuelt kan være substitueret med et eller flere halogenatomer; R7 er -OH; R6 er hydrogen; og R8 er hydrogen, forudsat at R2 og R3 ikke begge kan være hydrogen, når R4 er halogen.
3. Forbindelse valgt blandt:
eller et farmaceutisk acceptabelt salt eller solvat deraf eller en hvilken som helst kombination af to eller flere deraf; til anvendelse til forebyggelse og/eller behandling af en lysosomal ophobningssygdom hos en patient, som har risiko for at udvikle eller er diagnosticeret med en sådan sygdom.
4. Forbindelse til anvendelse ifølge krav 3, hvor forbindelsen er:
eller et farmaceutisk acceptabelt salt eller solvat deraf.
5. Forbindelse til anvendelse ifølge krav 3, hvor forbindelsen er:
eller et farmaceutisk acceptabelt salt eller solvat deraf.
6. Forbindelse til anvendelse ifølge krav 3, hvor forbindelsen er:
eller et farmaceutisk acceptabelt salt eller solvat deraf.
7. Forbindelse til anvendelse ifølge krav 3, hvor forbindelsen er:
eller et farmaceutisk acceptabelt salt eller solvat deraf.
8. Forbindelse til anvendelse ifølge krav 3, hvor forbindelsen er:
eller et farmaceutisk acceptabelt salt eller solvat deraf.
9. Forbindelse til anvendelse ifølge et hvilket som helst af de foregående krav, hvor den lysosomale ophobningssygdom er Niemann-Picks sygdom.
10. Forbindelse til anvendelse ifølge et hvilket som helst af kravene 1-8, hvor den lysosomale ophobningssygdom er Gauchers sygdom.
11. Forbindelse til anvendelse ifølge et hvilket som helst af kravene 1-10 i kombination med mindst ét andet terapeutisk middel.
12. Forbindelse til anvendelse ifølge 11, hvor mindst ét andet terapeutisk middel er imiglucerase eller 1,5-(butylimino)-1,5-dideoxy-D-glucitol.
13. Kit, som omfatter: • en beholder med en effektiv mængde af en forbindelse med formlen:
j eller et farmaceutisk acceptabelt salt eller solvat deraf; og • anvisninger vedrørende brug deraf til at forebygge eller behandle en lysosomal ophobningssygdom.
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US25280609P | 2009-10-19 | 2009-10-19 | |
| PCT/US2010/051458 WO2011049737A1 (en) | 2009-10-19 | 2010-10-05 | Novel compositions for preventing and/or treating lysosomal storage disorders |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| DK2490533T3 true DK2490533T3 (da) | 2015-12-07 |
Family
ID=43879464
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| DK10825397.2T DK2490533T3 (da) | 2009-10-19 | 2010-10-05 | Hidtil ukendte sammensætninger til forebyggelse og/eller behandling af lysosomale ophobningssygdomme |
| DK15183960.2T DK2995306T3 (da) | 2009-10-19 | 2010-10-05 | Hidtil ukendte sammensætninger til forebyggelse og/eller behandling af lysosomale aflejringssygdomme |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| DK15183960.2T DK2995306T3 (da) | 2009-10-19 | 2010-10-05 | Hidtil ukendte sammensætninger til forebyggelse og/eller behandling af lysosomale aflejringssygdomme |
Country Status (26)
| Country | Link |
|---|---|
| US (3) | US8604057B2 (da) |
| EP (2) | EP2995306B1 (da) |
| JP (3) | JP6147005B2 (da) |
| KR (1) | KR101848938B1 (da) |
| CN (2) | CN102647905B (da) |
| AR (1) | AR078607A1 (da) |
| AU (2) | AU2010308397B2 (da) |
| BR (1) | BR112012009123A2 (da) |
| CA (1) | CA2778349C (da) |
| DK (2) | DK2490533T3 (da) |
| ES (2) | ES2716871T3 (da) |
| HK (2) | HK1218082A1 (da) |
| HR (2) | HRP20151279T1 (da) |
| HU (2) | HUE028129T2 (da) |
| IL (1) | IL219231A0 (da) |
| LT (1) | LT2995306T (da) |
| MX (1) | MX337933B (da) |
| PL (2) | PL2995306T3 (da) |
| PT (2) | PT2995306T (da) |
| RS (2) | RS58466B1 (da) |
| RU (2) | RU2608520C2 (da) |
| SI (2) | SI2995306T1 (da) |
| SM (1) | SMT201500280B (da) |
| TR (1) | TR201903901T4 (da) |
| TW (2) | TWI558396B (da) |
| WO (1) | WO2011049737A1 (da) |
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| WO2001056596A1 (en) * | 2000-02-04 | 2001-08-09 | Children's Hospital Research Foundation | Use of lysosomal acid lipase for treating atherosclerosis and related diseases |
| US8889127B2 (en) | 2004-07-01 | 2014-11-18 | Icahn School Of Medicine At Mount Sinai | Targeted protein replacement for the treatment of lysosomal storage disorders |
| US20090297496A1 (en) * | 2005-09-08 | 2009-12-03 | Childrens Hospital Medical Center | Lysosomal Acid Lipase Therapy for NAFLD and Related Diseases |
| PT2995306T (pt) | 2009-10-19 | 2019-03-29 | Amicus Therapeutics Inc | Novas composições para prevenção e/ou tratamento de disfunções de armazenamento lisossomal |
| ES2814178T3 (es) | 2009-10-19 | 2021-03-26 | Amicus Therapeutics Inc | Nuevas composiciones para prevenir y/o tratar trastornos degenerativos del sistema nervioso central |
| PT3205351T (pt) | 2010-04-23 | 2023-06-28 | Alexion Pharma Inc | Enzima de doença de armazenamento lisossomal |
| CN105457018A (zh) | 2010-09-09 | 2016-04-06 | 辛那杰瓦生物制药股份有限公司 | 使用溶酶体酸性脂肪酶来治疗患者的溶酶体酸性脂肪酶缺乏 |
| WO2012112681A1 (en) | 2011-02-15 | 2012-08-23 | Shire Human Genetic Therapies, Inc. | Methods for treating lysosomal acid lipase deficiency |
| ES2664844T3 (es) | 2011-06-20 | 2018-04-23 | Mount Sinai School Of Medicine | Tratamiento dirigido contra TNF para las mucopolisacaridosis y otros trastornos lisosómicos |
| WO2013036875A1 (en) | 2011-09-07 | 2013-03-14 | Mount Sinai School Of Medicine | Ceramidase and cell differentiation |
| AU2013240486B2 (en) * | 2012-03-27 | 2017-10-26 | Amicus Therapeutics, Inc. | Novel compounds for preventing and/or treating lysosomal storage disorders and/or degenerative disorders of the central nervous system |
| CA2874146C (en) | 2012-06-01 | 2023-03-21 | Icahn School Of Medicine At Mount Sinai | Ceramide levels in the treatment and prevention of infections |
| WO2014160390A1 (en) | 2013-03-14 | 2014-10-02 | Icahn School Of Medicine At Mount Sinai | Therapeutic acid ceramidase compositions and methods of making and using them |
| EP3087056A4 (en) | 2013-12-23 | 2017-07-19 | Alectos Therapeutics Inc. | Glucocerebrosidase modulators and uses thereof |
| WO2015123385A1 (en) * | 2014-02-12 | 2015-08-20 | Amicus Therapeutics, Inc. | Sugar derivatives comprising sulfur-containing moieties and methods of making same and methods of using the same for the treatment of mps iiic |
| EP3220906B1 (en) | 2014-11-19 | 2022-03-02 | Rush University Medical Center | Compositions and methods for treating lysosomal disorders |
| EP3088389A1 (en) * | 2015-04-28 | 2016-11-02 | Dorphan S.A. | 4-epi-isofagomine derivatives |
| US10179128B2 (en) * | 2015-08-31 | 2019-01-15 | Amicus Therapeutics, Inc. | Regimens for treating and preventing lysosomal disorders and degenerative disorders of the central nervous system |
| AR107483A1 (es) | 2016-01-29 | 2018-05-02 | Hanmi Pharm Ind Co Ltd | Conjugado de enzimas terapéuticas |
| BR112019013593A2 (pt) | 2016-12-28 | 2020-01-07 | Minoryx Therapeutics S.L. | Compostos de isoquinolina, métodos para sua preparação, e usos terapêuticos dos mesmos em afecções associadas à alteração da atividade de beta galactosidase |
| US11174242B2 (en) | 2016-12-29 | 2021-11-16 | Minoryx Therapeutics S.L. | Heteroaryl compounds and their use |
| CN108524952A (zh) * | 2017-03-06 | 2018-09-14 | 复旦大学 | 腺相关病毒aav9及其在制备治疗戈谢氏病的制剂中的用途 |
| AU2018258348B2 (en) | 2017-04-25 | 2022-07-07 | Amicus Therapeutics, Inc. | Novel compositions for preventing and/or treating degenerative disorders of the central nervous system and/or lysosomal storage disorders |
| CN108727249A (zh) * | 2018-07-07 | 2018-11-02 | 台州学院 | 3-二氟甲基哌啶盐酸盐及其衍生物的合成方法 |
| US20220184185A1 (en) | 2018-07-25 | 2022-06-16 | Modernatx, Inc. | Mrna based enzyme replacement therapy combined with a pharmacological chaperone for the treatment of lysosomal storage disorders |
| US20220016105A1 (en) * | 2018-11-13 | 2022-01-20 | EDWARD Via COLLEGE OF OSTEOPATHIC MEDICINE | Biased nmda receptor modulators and uses thereof |
| JP2023503465A (ja) | 2019-11-25 | 2023-01-30 | ゲイン セラピューティクス エスアー | ベータ－グルコセレブロシダーゼの活性の変質に関連する状態におけるヘテロアリール化合物およびその治療的使用 |
| BR112022010127A2 (pt) | 2019-11-25 | 2022-09-06 | Gain Therapeutics Sa | Compostos de arila e heteroarila e usos terapêuticos dos mesmos em condições associadas à alteração da atividade de galatocerebrosidase |
| JP2023512281A (ja) | 2020-02-03 | 2023-03-24 | ゲイン セラピューティクス エスアー | Ｍｐｓ１を処置するための併用療法 |
| WO2022212271A1 (en) * | 2021-03-29 | 2022-10-06 | Sanford Health | Methods and compositions for treating lysosomal storage disorders |
| WO2023042177A1 (en) | 2021-09-20 | 2023-03-23 | Gt Gain Therapeutics Sa | Enantiomers of 5-((7-chloroisoquinolin-1-yl)amino)-n-(6-methoxy-1,2,3,4-tetrahydronaphthalen-2-yl)picolinamide |
| WO2024141998A1 (en) | 2022-12-30 | 2024-07-04 | Gt Gain Therapeutics Sa | Method of treating tauopathies |
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