BICALUTAMIDE ANALOGS OR (S)-BICALUTAMIDE AS EXOCYTOSIS ACTIVATING COMPOUNDS FOR USE IN THE TREATMENT OF A LYSOSOMAL STORAGE DISORDER OR GLYCOGENOSIS
The invention provides a therapy for lysosomal storage diseases and glycogenosis by treatment with compounds that promote exocytosis, preferably lysosomal exocytosis. The treatment of cells from patients affected by different lysosomal storage disorders with exocytosis activating compounds leads to...
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| Main Authors | , , , , , , |
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| Format | Patent |
| Language | English Hungarian |
| Published |
30.09.2019
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| Subjects | |
| Online Access | Get full text |
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| Abstract | The invention provides a therapy for lysosomal storage diseases and glycogenosis by treatment with compounds that promote exocytosis, preferably lysosomal exocytosis. The treatment of cells from patients affected by different lysosomal storage disorders with exocytosis activating compounds leads to a decrease in the accumulation of toxic substrate in the lysosomes, thus allowing the treatment, prevention and relief of the symptoms of many lysosomal storage disorders. |
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| AbstractList | The invention provides a therapy for lysosomal storage diseases and glycogenosis by treatment with compounds that promote exocytosis, preferably lysosomal exocytosis. The treatment of cells from patients affected by different lysosomal storage disorders with exocytosis activating compounds leads to a decrease in the accumulation of toxic substrate in the lysosomes, thus allowing the treatment, prevention and relief of the symptoms of many lysosomal storage disorders. |
| Author | RIBES RUBIO, Antonia PONSATI OBIOLS, Berta FARRERA-SINFREU, Josep PASCUAL MARTINEZ, Roberto GORT MAS, Laura FERRER MONTIEL, Antonio MATALONGA BORREL, Leslie |
| Author_xml | – fullname: PONSATI OBIOLS, Berta – fullname: PASCUAL MARTINEZ, Roberto – fullname: RIBES RUBIO, Antonia – fullname: MATALONGA BORREL, Leslie – fullname: FERRER MONTIEL, Antonio – fullname: FARRERA-SINFREU, Josep – fullname: GORT MAS, Laura |
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| DocumentTitleAlternate | Bikalutamid analógok vagy (S)-bikalutamid, mint exocitózist aktiváló vegyületek lizoszomális tárolási zavar vagy glikogenózis kezelésében történõ alkalmazásra |
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| Snippet | The invention provides a therapy for lysosomal storage diseases and glycogenosis by treatment with compounds that promote exocytosis, preferably lysosomal... |
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| Title | BICALUTAMIDE ANALOGS OR (S)-BICALUTAMIDE AS EXOCYTOSIS ACTIVATING COMPOUNDS FOR USE IN THE TREATMENT OF A LYSOSOMAL STORAGE DISORDER OR GLYCOGENOSIS |
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