SLS-005 (seaweed sugar) treatment of Sanfilippo syndrome: EMA takes a positive view.
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Last Update: 2020-07-28
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Source: Internet
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Author: User
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Sticky polysaccharides are lysosome synthases that occur due to the lack of enzymes needed to gradually degrade glycoamine polysaccharides ()Partially degraded fragments accumulate in lysozyme, leading to cell dysfunction and clinical abnormalitiesType of mucosal polysaccharide disease is clinically visible for children in the age of normal development after the gradual emergence of language, behavioral disorders growth and backwardness in childhood nervous system degenerative lesions are more obvious liver and spleen enlargement, argon, ugly face, joint stiffness and other performanceA clinically phased biopharmaceutical company dedicated to the development of therapies for rare diseases involving central nervous system diseases today announced that it has received a positive evaluation of the qualifications of orphan drugs targeted by the European Medicines Agency () Orphan Medicines Commission ()This year was awarded the title of orphan medicine for the treatment of the syndrome in the United StatesPreviously awarded and treated for spinal cord microcephaly disorder () and opharynx muscular dystrophy () orphan drug designationAlso qualified for fast track
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