(II).;EFFECT: invention also relates to use of said compounds in diagnosing diseases and/or disorders of lysosomal accumulation and/or glycogenosis and using a pharmaceutical composition containing said compounds.;11 cl, 1 dwg, 5 tbl, 6 ex"/> ANALOGUES OF BICALUTAMIDE OR (S)-BICALUTAMIDE AS EXOCYTOSIS ACTIVATING COMPOUNDS FOR USE IN TREATMENT OF LYSOSOMAL STORAGE DISORDER OR GLYCOGENOSIS
首页> 外国专利> ANALOGUES OF BICALUTAMIDE OR (S)-BICALUTAMIDE AS EXOCYTOSIS ACTIVATING COMPOUNDS FOR USE IN TREATMENT OF LYSOSOMAL STORAGE DISORDER OR GLYCOGENOSIS

ANALOGUES OF BICALUTAMIDE OR (S)-BICALUTAMIDE AS EXOCYTOSIS ACTIVATING COMPOUNDS FOR USE IN TREATMENT OF LYSOSOMAL STORAGE DISORDER OR GLYCOGENOSIS

机译:比卡洛胺或(S)-比卡洛胺的类似物作为促溶酶活性化合物用于溶酶体贮积病或糖原异生的治疗

摘要

FIELD: medicine.;SUBSTANCE: invention relates to use of compound (S)-bicalutamide and/or structural analogue, which are collectively determined by general formula (II), as well as their pharmaceutically acceptable salts, hydrates and/or solvates for preventing clinical symptoms and/or treatment of diseases and/or disorders of lysosomal accumulation and/or glycogenosis. In formula (II) R1 is halogen (F, Cl, Br or I), R4 is a trifluoromethyl group (-CF3), R5 is a nitrile (-CN) group, X is a thioether (S), a sulphoxide (SO), a sulphone (SO2) or oxygen (O). (II).;EFFECT: invention also relates to use of said compounds in diagnosing diseases and/or disorders of lysosomal accumulation and/or glycogenosis and using a pharmaceutical composition containing said compounds.;11 cl, 1 dwg, 5 tbl, 6 ex
机译:技术领域本发明涉及由通式(II)共同确定的化合物(S)-比卡鲁胺和/或结构类似物及其药学上可接受的盐,水合物和/或溶剂化物的用途。溶酶体蓄积和/或糖原异生的疾病和/或疾病的临床症状和/或治疗。在式(II)中,R 1 是卤素(F,Cl,Br或I),R 4 是三氟甲基(-CF 3 ),R 5 是腈(-CN)基团,X是硫醚(S),亚砜(SO),砜(SO 2 )或氧( O)。 <图像文件=“ 00000046.JPG” he =“ 55” imgContent =“ undefined” imgFormat =“ JPEG” wi =“ 48” />(II)。效果:本发明还涉及所述化合物在诊断疾病和疾病中的用途。和/或溶酶体蓄积和/或糖原异生疾病,并使用含有所述化合物的药物组合物。11cl,1 dwg,5 tbl,6 ex

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