Bicalutamide Analogs Or (S)-Bicalutamide As Exocytosis Activating Compounds For Use In The Treatment Of A Lysosomal Storage Disorder Or Glycogenosis
SUMMARY 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 l...
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| creator | GORT MAS Laura FARRERA-SINFREU Josep PONSATI OBIOLS Berta RIBES RUBIÓ Antonia MATALONGA BORREL Leslie FERRER MONTIEL Antonio PASCUAL MARTÍNEZ Roberto |
| description | SUMMARY 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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| subjects | HUMAN NECESSITIES HYGIENE INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIRCHEMICAL OR PHYSICAL PROPERTIES MEASURING MEDICAL OR VETERINARY SCIENCE PHYSICS PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES TESTING |
| 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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