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
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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
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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
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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
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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
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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
- 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
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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
- A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
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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
- A61P1/18—Drugs for disorders of the alimentary tract or the digestive system for pancreatic disorders, e.g. pancreatic enzymes
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- 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
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- 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
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- 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
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- 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
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- 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
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- 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
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- 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
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- 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
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- 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
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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
- C07D211/36—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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- 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)
- Public Health (AREA)
- Medicinal Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Engineering & Computer Science (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Epidemiology (AREA)
- Diabetes (AREA)
- Hematology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Immunology (AREA)
- Obesity (AREA)
- Neurosurgery (AREA)
- Physical Education & Sports Medicine (AREA)
- Neurology (AREA)
- Dermatology (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)
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 |
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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) | EP2490533B1 (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) | DK2995306T3 (es) |
ES (2) | ES2555533T3 (es) |
HK (2) | 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) | RU2016103139A (es) |
SI (2) | SI2490533T1 (es) |
SM (1) | SMT201500280B (es) |
TR (1) | TR201903901T4 (es) |
TW (2) | TWI587859B (es) |
WO (1) | WO2011049737A1 (es) |
Families Citing this family (31)
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AU2010308397B2 (en) | 2009-10-19 | 2016-04-07 | Amicus Therapeutics, Inc. | Novel compositions for preventing and/or treating lysosomal storage disorders |
RS55679B1 (sr) | 2009-10-19 | 2017-07-31 | Amicus Therapeutics Inc | Nove smeše za sprečavanje i/ili lečenje degenerativnih poremećaja centralnog nervnog sistema |
ES2950358T3 (es) | 2010-04-23 | 2023-10-09 | Alexion Pharma Inc | Enzima de la enfermedad de almacenamiento lisosomal |
JP5693728B2 (ja) | 2010-09-09 | 2015-04-01 | シナジーバ バイオファーマ コープ | 患者のリソソーム酸リパーゼ欠乏症を治療するためのリソソーム酸リパーゼの使用 |
KR20140006037A (ko) | 2011-02-15 | 2014-01-15 | 시나게바 바이오파르마, 코포레이션 | 리소좀의 산 리파제 결핍을 치료하는 방법들 |
WO2012177778A1 (en) * | 2011-06-20 | 2012-12-27 | Mount Sinai School Of Medicine | Anti-tnf- therapy for the mucopolysaccharidoses and other lysosomal disorders |
ES2791760T3 (es) | 2011-09-07 | 2020-11-05 | Sinai School Medicine | Ceramidasa y diferenciación celular |
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 |
LT2854910T (lt) | 2012-06-01 | 2020-05-25 | Icahn School Of Medicine At Mount Sinai | Ceramido lygiai infekcijos gydymo ir prevencijos metu |
PT2968479T (pt) | 2013-03-14 | 2019-08-07 | Icahn School Med Mount Sinai | Composições terapêuticas de ceramidase ácida e métodos de fabrico e utilização |
EP3087056A4 (en) | 2013-12-23 | 2017-07-19 | Alectos Therapeutics Inc. | Glucocerebrosidase modulators and uses thereof |
KR20160113720A (ko) * | 2014-02-12 | 2016-09-30 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 황 함유 모이어티를 포함하는 당 유도체 및 이의 제조 방법 및 mps iiic의 치료를 위한 이의 사용 방법 |
ES2914085T3 (es) * | 2014-11-19 | 2022-06-07 | Univ Rush Medical Center | Composiciones y métodos para el tratamiento de trastornos lisosomales |
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 |
ES2862374T3 (es) | 2016-12-28 | 2021-10-07 | Minoryx Therapeutics S L | Compuestos de isoquinolina, métodos para su preparación y usos terapéuticos de los mismos en afecciones asociadas con la alteración de la actividad de beta galactosidasa |
EP3562811B1 (en) | 2016-12-29 | 2022-03-16 | 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 |
WO2018200618A1 (en) * | 2017-04-25 | 2018-11-01 | 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 |
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WO2020102443A1 (en) * | 2018-11-13 | 2020-05-22 | 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 |
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