CN107205976A - 用于治疗溶酶体病症的组合物和方法 - Google Patents

用于治疗溶酶体病症的组合物和方法 Download PDF

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Publication number
CN107205976A
CN107205976A CN201580073947.8A CN201580073947A CN107205976A CN 107205976 A CN107205976 A CN 107205976A CN 201580073947 A CN201580073947 A CN 201580073947A CN 107205976 A CN107205976 A CN 107205976A
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disease
tfeb
pparα
lysosomal
aspirin
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Chinese (zh)
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K·帕汉
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Rush University Medical Center
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Rush University Medical Center
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/60—Salicylic acid; Derivatives thereof
    • A61K31/612—Salicylic acid; Derivatives thereof having the hydroxy group in position 2 esterified, e.g. salicylsulfuric acid
    • A61K31/616—Salicylic acid; Derivatives thereof having the hydroxy group in position 2 esterified, e.g. salicylsulfuric acid by carboxylic acids, e.g. acetylsalicylic acid
    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/045—Hydroxy compounds, e.g. alcohols; Salts thereof, e.g. alcoholates
    • A61K31/07—Retinol compounds, e.g. vitamin A
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    • A61K31/185—Acids; Anhydrides, halides or salts thereof, e.g. sulfur acids, imidic, hydrazonic or hydroximic acids
    • A61K31/19—Carboxylic acids, e.g. valproic acid
    • A61K31/192—Carboxylic acids, e.g. valproic acid having aromatic groups, e.g. sulindac, 2-aryl-propionic acids, ethacrynic acid 
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    • A61K31/19—Carboxylic acids, e.g. valproic acid
    • A61K31/20—Carboxylic acids, e.g. valproic acid having a carboxyl group bound to a chain of seven or more carbon atoms, e.g. stearic, palmitic, arachidic acids
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    • A61K31/215—Esters, e.g. nitroglycerine, selenocyanates of carboxylic acids
    • A61K31/216—Esters, e.g. nitroglycerine, selenocyanates of carboxylic acids of acids having aromatic rings, e.g. benactizyne, clofibrate
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    • A61K31/215—Esters, e.g. nitroglycerine, selenocyanates of carboxylic acids
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    • A61K31/215—Esters, e.g. nitroglycerine, selenocyanates of carboxylic acids
    • A61K31/22—Esters, e.g. nitroglycerine, selenocyanates of carboxylic acids of acyclic acids, e.g. pravastatin
    • A61K31/222—Esters, e.g. nitroglycerine, selenocyanates of carboxylic acids of acyclic acids, e.g. pravastatin with compounds having aromatic groups, e.g. dipivefrine, ibopamine
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K31/33—Heterocyclic compounds
    • A61K31/335—Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin
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    • A61K31/366—Lactones having six-membered rings, e.g. delta-lactones
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    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/40—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with one nitrogen as the only ring hetero atom, e.g. sulpiride, succinimide, tolmetin, buflomedil
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/40—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with one nitrogen as the only ring hetero atom, e.g. sulpiride, succinimide, tolmetin, buflomedil
    • A61K31/403—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with one nitrogen as the only ring hetero atom, e.g. sulpiride, succinimide, tolmetin, buflomedil condensed with carbocyclic rings, e.g. carbazole
    • A61K31/404—Indoles, e.g. pindolol
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    • A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/505—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
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    • A61P21/02—Muscle relaxants, e.g. for tetanus or cramps
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
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    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
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    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00

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  • Pharmacology & Pharmacy (AREA)
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  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurology (AREA)
  • Biomedical Technology (AREA)
  • Neurosurgery (AREA)
  • Emergency Medicine (AREA)
  • Psychology (AREA)
  • Hospice & Palliative Care (AREA)
  • Diabetes (AREA)
  • Psychiatry (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Urology & Nephrology (AREA)
  • Pain & Pain Management (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
CN201580073947.8A 2014-11-19 2015-11-16 用于治疗溶酶体病症的组合物和方法 Pending CN107205976A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201462081696P 2014-11-19 2014-11-19
US62/081,696 2014-11-19
PCT/US2015/060878 WO2016081365A1 (en) 2014-11-19 2015-11-16 Compositions and methods for treating lysosomal disorders

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Publication Number Publication Date
CN107205976A true CN107205976A (zh) 2017-09-26

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US (3) US20170354666A1 (https=)
EP (2) EP4026545A1 (https=)
JP (4) JP2017536363A (https=)
KR (1) KR20170083146A (https=)
CN (1) CN107205976A (https=)
AU (1) AU2015350223B2 (https=)
CA (2) CA3176253A1 (https=)
ES (1) ES2914085T3 (https=)
WO (1) WO2016081365A1 (https=)

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KR20210143848A (ko) * 2019-03-22 2021-11-29 러쉬 유니버시티 메디컬 센터 리소좀 축적 장애를 위한 비강 유전자 전달 및 경구 신남산, 올레아미드 또는 젬피브로질의 병용
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CN116723870A (zh) * 2021-01-20 2023-09-08 拉什大学医学中心 球形细胞脑白质营养不良症或克拉伯病的改良治疗
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US20230037062A1 (en) 2023-02-02
JP2022003060A (ja) 2022-01-11
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WO2016081365A1 (en) 2016-05-26
AU2015350223A1 (en) 2017-05-11
EP3220906A4 (en) 2018-04-25
KR20170083146A (ko) 2017-07-17
US20250134911A1 (en) 2025-05-01
US20170354666A1 (en) 2017-12-14
CA2967066A1 (en) 2016-05-26
EP3220906A1 (en) 2017-09-27
EP4026545A1 (en) 2022-07-13
ES2914085T3 (es) 2022-06-07
JP2020100621A (ja) 2020-07-02
JP2021107404A (ja) 2021-07-29
US12023345B2 (en) 2024-07-02
CA2967066C (en) 2026-01-13
JP2017536363A (ja) 2017-12-07
CA3176253A1 (en) 2016-05-26
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