AR078607A1 - Composiciones para prevenir y/ o tratar trastornos de almacenamiento lisosomal - Google Patents
Composiciones para prevenir y/ o tratar trastornos de almacenamiento lisosomalInfo
- Publication number
- AR078607A1 AR078607A1 ARP100103716A ARP100103716A AR078607A1 AR 078607 A1 AR078607 A1 AR 078607A1 AR P100103716 A ARP100103716 A AR P100103716A AR P100103716 A ARP100103716 A AR P100103716A AR 078607 A1 AR078607 A1 AR 078607A1
- Authority
- AR
- Argentina
- Prior art keywords
- substituted
- alkyl
- hydrogen
- halogen
- compound
- Prior art date
Links
- 239000000203 mixture Substances 0.000 title abstract 2
- 125000004209 (C1-C8) alkyl group Chemical group 0.000 abstract 13
- 238000000034 method Methods 0.000 abstract 10
- 229910052739 hydrogen Inorganic materials 0.000 abstract 9
- 239000001257 hydrogen Substances 0.000 abstract 9
- 229910052736 halogen Inorganic materials 0.000 abstract 7
- 150000001875 compounds Chemical class 0.000 abstract 6
- 150000002367 halogens Chemical class 0.000 abstract 5
- 150000002431 hydrogen Chemical group 0.000 abstract 5
- 239000000651 prodrug Substances 0.000 abstract 5
- 229940002612 prodrug Drugs 0.000 abstract 5
- 150000003839 salts Chemical class 0.000 abstract 5
- 239000012453 solvate Substances 0.000 abstract 5
- 125000002877 alkyl aryl group Chemical group 0.000 abstract 4
- 125000003710 aryl alkyl group Chemical group 0.000 abstract 4
- 208000015439 Lysosomal storage disease Diseases 0.000 abstract 3
- UFHFLCQGNIYNRP-UHFFFAOYSA-N Hydrogen Chemical compound [H][H] UFHFLCQGNIYNRP-UHFFFAOYSA-N 0.000 abstract 2
- 125000003342 alkenyl group Chemical group 0.000 abstract 2
- 125000005119 alkyl cycloalkyl group Chemical group 0.000 abstract 2
- 125000003118 aryl group Chemical group 0.000 abstract 2
- 125000005843 halogen group Chemical group 0.000 abstract 2
- 125000004435 hydrogen atom Chemical group [H]* 0.000 abstract 2
- 125000003107 substituted aryl group Chemical group 0.000 abstract 2
- -1 -OH Inorganic materials 0.000 abstract 1
- OKTJSMMVPCPJKN-UHFFFAOYSA-N Carbon Chemical group [C] OKTJSMMVPCPJKN-UHFFFAOYSA-N 0.000 abstract 1
- 208000015872 Gaucher disease Diseases 0.000 abstract 1
- 102100033342 Lysosomal acid glucosylceramidase Human genes 0.000 abstract 1
- 208000014060 Niemann-Pick disease Diseases 0.000 abstract 1
- 229910052799 carbon Inorganic materials 0.000 abstract 1
- 125000000753 cycloalkyl group Chemical group 0.000 abstract 1
- 239000003814 drug Substances 0.000 abstract 1
- 125000001153 fluoro group Chemical group F* 0.000 abstract 1
- 229960002127 imiglucerase Drugs 0.000 abstract 1
- 108010039650 imiglucerase Proteins 0.000 abstract 1
- 229940124597 therapeutic agent Drugs 0.000 abstract 1
Classifications
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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
-
- 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)
-
- 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
-
- 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
-
- 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
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- 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
- C07D211/04—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
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Life Sciences & Earth Sciences (AREA)
- Veterinary Medicine (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- General Health & Medical Sciences (AREA)
- Public Health (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Epidemiology (AREA)
- Diabetes (AREA)
- Hematology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Immunology (AREA)
- Obesity (AREA)
- Dermatology (AREA)
- Heart & Thoracic Surgery (AREA)
- Physical Education & Sports Medicine (AREA)
- Neurosurgery (AREA)
- Cardiology (AREA)
- Neurology (AREA)
- Biomedical Technology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Hydrogenated Pyridines (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Abstract
La presente proporciona composiciones, así como también métodos para prevenir y/o tratar trastornos de almacenamiento lisosomal. En particular, la presente proporciona métodos para prevenir y/o tratar la enfermedad de Gaucher. Reivindicacion 1: Un método para prevenir y/o tratar un trastorno de almacenamiento lisosomal en un paciente con riesgo de desarrollar o que se le diagnostique el mismo, que comprende administrar al paciente que lo necesita una cantidad efectiva de un compuesto de formula (1) en donde R1 es C(R2)(R3)(R4); R2 es hidrogeno, -OH o halogeno; R3 es hidrogeno, -OH, halogeno o alquilo C1-8; R4 es halogeno, alquilo C1-8, alquilo C1-8 sustituido, arilo, arilo sustituido, alquilcicloalquilo o alquilcicloalquilo sustituido; R3 y R4 pueden juntarse con el carbono al que están unidos para formar un anillo cicloalquilo, que puede estar opcionalmente sustituido, preferiblemente por halogeno y más preferiblemente por uno o más átomos de fluor; R6 es hidrogeno, alquilo C1-8, alquilo C1-8 sustituido, arilalquilo, arilalquilo sustituido, alquilarilo o alquilarilo sustituido; Z es opcional, cuando está presente Z es -(CH2)1-8-, -C(=O)-, -S(=O)2NH-, -S(=O)2-, -C(=S)NH-, -S(=O)2-CH3, C(=O)-NH-, -S(=O)2-NR9R10, - C(=O)alquilo C1-8 o -C(=O)CH(NH2)CH3; R9 es hidrogeno, alquilo C1-8 o alquilo C1-8 sustituido; R10 es hidrogeno, alquilo C1-8 o alquilo C1-8 sustituido; R5 es hidrogeno, alquilo C1-8, alquilo C1-8 sustituido, arilo, arilo sustituido, alquenilo C1-8, alquenilo C1-8 sustituido, arilalquilo, arilalquilo sustituido, alquilarilo, alquilarilo sustituido, aminoarilalquilo o aminoarilalquilo sustituido; R7 es -OH o halogeno; y R8 es hidrogeno, halogeno o alquilo C1-8; siempre que R2 y R3 no sean ambos hidrogeno cuando R4 sea un halogeno, Z no esté presente, R7 sea -OH, R5, R6 y R8 sean hidrogeno. Reivindicacion 5: El método de la reivindicacion 4, en donde el compuesto es como en formula (2) o una sal, solvato o profármaco farmacéuticamente aceptable del mismo. Reivindicacion 6: El método de la reivindicacion 4, en donde el compuesto es como en formula (3) o una sal, solvato o profármaco farmacéuticamente aceptable del mismo. Reivindicacion 7: El método de la reivindicacion 4, en donde el compuesto es como en formula (4) o una sal, solvato o profármaco farmacéuticamente aceptable del mismo. Reivindicacion 8: El método de la reivindicacion 4, en donde el compuesto es como en formula (5) o una sal, solvato o profármaco farmacéuticamente aceptable del mismo. Reivindicacion 9: El método de la reivindicacion 4, en donde el compuesto es como en formula (6) o una sal, solvato o profármaco farmacéuticamente aceptable del mismo. Reivindicacion 11: El método de la reivindicacion 1, en donde la enfermedad de almacenamiento lisosomal es la enfermedad de Niemann-Pick. Reivindicacion 14: El método de la reivindicacion 13, en donde al menos otro agente terapéutico es imiglucerasa o 1,5-(butilimino)-1,5-didesoxi-D-glucitol.
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US25280609P | 2009-10-19 | 2009-10-19 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| AR078607A1 true AR078607A1 (es) | 2011-11-23 |
Family
ID=43879464
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ARP100103716A AR078607A1 (es) | 2009-10-19 | 2010-10-12 | Composiciones para prevenir y/ o tratar trastornos de almacenamiento lisosomal |
Country Status (26)
| Country | Link |
|---|---|
| US (3) | US8604057B2 (es) |
| EP (2) | EP2995306B1 (es) |
| JP (3) | JP6147005B2 (es) |
| KR (1) | KR101848938B1 (es) |
| CN (2) | CN105193802A (es) |
| AR (1) | AR078607A1 (es) |
| AU (2) | AU2010308397B2 (es) |
| BR (1) | BR112012009123A2 (es) |
| CA (1) | CA2778349C (es) |
| DK (2) | DK2490533T3 (es) |
| ES (2) | ES2555533T3 (es) |
| HK (1) | HK1218082A1 (es) |
| HR (2) | HRP20151279T1 (es) |
| HU (2) | HUE041971T2 (es) |
| IL (1) | IL219231A0 (es) |
| LT (1) | LT2995306T (es) |
| MX (1) | MX337933B (es) |
| PL (2) | PL2995306T4 (es) |
| PT (2) | PT2995306T (es) |
| RS (2) | RS58466B1 (es) |
| RU (2) | RU2608520C2 (es) |
| SI (2) | SI2490533T1 (es) |
| SM (1) | SMT201500280B (es) |
| TR (1) | TR201903901T4 (es) |
| TW (2) | TWI558396B (es) |
| WO (1) | WO2011049737A1 (es) |
Families Citing this family (34)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6849257B2 (en) * | 2000-02-04 | 2005-02-01 | Children's Hospital Research Foundation | Lipid hydrolysis therapy for 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 |
| WO2007030375A2 (en) * | 2005-09-08 | 2007-03-15 | Children's Hospital Medical Center | Lysosomal acid lipase therapy for nafld and related diseases |
| WO2011049737A1 (en) * | 2009-10-19 | 2011-04-28 | Amicus Therapeutics, Inc. | Novel compositions for preventing and/or treating lysosomal storage disorders |
| KR101880411B1 (ko) | 2009-10-19 | 2018-07-19 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 중추신경계의 퇴행성 질환을 예방 및/또는 치료하는 신규 조성물 |
| MX338426B (es) | 2010-04-23 | 2016-04-15 | Synageva Biopharma Corp | Enzima de enfermedad del almacenamiento lisosomico. |
| PL2613798T5 (pl) | 2010-09-09 | 2018-06-29 | Synageva Biopharma Corp. | Zastosowanie lizosomalnej kwaśnej lipazy do leczenia niedoboru lizosomalnej kwaśnej lipazy u pacjentów |
| EP2675472A4 (en) | 2011-02-15 | 2014-09-17 | Synageva Biopharma Corp | METHOD FOR THE TREATMENT OF LACK OF LYSOSOMAL SAURER LIPASE |
| EP2721151B1 (en) | 2011-06-20 | 2017-12-06 | Mount Sinai School Of Medicine | Anti-tnf-therapy for the mucopolysaccharidoses and other lysosomal disorders |
| WO2013036875A1 (en) | 2011-09-07 | 2013-03-14 | Mount Sinai School Of Medicine | Ceramidase and cell differentiation |
| HK1205503A1 (en) | 2012-03-27 | 2015-12-18 | Amicus Therapeutics, Inc. | Novel compounds for preventing and/or treating lysosomal storage disorders and/or degenerative disorders of the central nervous system |
| EP3679923B1 (en) | 2012-06-01 | 2021-08-04 | Icahn School of Medicine at Mount Sinai | Ceramide levels in the treatment and prevention of infections |
| DK2968479T3 (da) | 2013-03-14 | 2019-08-12 | Icahn School Med Mount Sinai | Terapeutiske sure ceramidasesammensætninger og fremgangsmåder til fremstilling og anvendelse heraf |
| EP3087056A4 (en) | 2013-12-23 | 2017-07-19 | Alectos Therapeutics Inc. | Glucocerebrosidase modulators and uses thereof |
| ES2771849T3 (es) | 2014-02-12 | 2020-07-07 | Amicus Therapeutics Inc | Derivados de azúcares que comprenden restos que contienen azufre, métodos para prepararlos y métodos para emplearlos en el tratamiento de MPS IIIC |
| WO2016081365A1 (en) * | 2014-11-19 | 2016-05-26 | 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 |
| WO2018122746A1 (en) | 2016-12-28 | 2018-07-05 | Minoryx Therapeutics S.L. | Isoquinoline compounds, methods for their preparation, and therapeutic uses thereof in conditions associated with the alteration of the activity of beta galactosidase |
| JP7171057B2 (ja) | 2016-12-29 | 2022-11-15 | ミノリックス セラピューティクス エセ．エレ． | ヘテロアリール化合物およびその使用 |
| 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 |
| KR20190134807A (ko) * | 2017-04-25 | 2019-12-04 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 중추 신경계의 퇴행성 질환 및/또는 리소솜 저장병을 예방 및/또는 치료하기 위한 신규한 조성물 |
| 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 |
| US12485115B2 (en) | 2018-11-13 | 2025-12-02 | EDWARD Via COLLEGE OF OSTEOPATHIC MEDICINE | Biased NMDA receptor modulators and uses thereof |
| JP2023503470A (ja) | 2019-11-25 | 2023-01-30 | ゲイン セラピューティクス エスアー | ガラクト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アリールおよびヘテロアリール化合物、ならびにそれらの治療的使用 |
| JP7691753B2 (ja) | 2019-11-25 | 2025-06-12 | ジーティー 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 |
| US20230140216A1 (en) | 2020-02-03 | 2023-05-04 | Gain Therapeutics Sa | Combination therapy for treating mps1 |
| WO2022212273A1 (en) * | 2021-03-29 | 2022-10-06 | Sanford Health | Methods and compositions for treating lysosomal storage disorders |
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