Search Results - "Therapeutic approach"
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Lysosomal storage diseases
Published 2019Table of Contents: “…Contents: Lysosomes are multifunctional -- Lysosome related organelles -- Complexity & functions of lysosomal proteins -- Lysosomal storage diseases -- Sphingolipid lysosomal storage diseases as an example -- Lysosomal diseases: multimorbidity disorders -- Pathogenic cascade beyond the Lysosomal System -- Specific therapies approved for some diseases -- Molecular mechanisms and therapeutic approaches of LSDs -- Lysosomal enzymes -- ERTs: approved or in advanced clinical trials -- Approved small molecules: SRT & EET -- Multiple gene therapy trials: enzyme deficiencies -- Cellular organelles targeted by LSD therapies -- Lysosomal & neurodegenerative diseases.…”
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Electronic Video -
2
Lysosomal storage diseases
Published 2019Table of Contents: “…Contents: Lysosomes are multifunctional -- Lysosome related organelles -- Complexity & functions of lysosomal proteins -- Lysosomal storage diseases -- Sphingolipid lysosomal storage diseases as an example -- Lysosomal diseases: multimorbidity disorders -- Pathogenic cascade beyond the Lysosomal System -- Specific therapies approved for some diseases -- Molecular mechanisms and therapeutic approaches of LSDs -- Lysosomal enzymes -- ERTs: approved or in advanced clinical trials -- Approved small molecules: SRT & EET -- Multiple gene therapy trials: enzyme deficiencies -- Cellular organelles targeted by LSD therapies -- Lysosomal & neurodegenerative diseases.…”
Get full text
Series
Electronic Video -
3
Lysosomal storage diseases
Published 2019Table of Contents: “…Contents: Lysosomes are multifunctional -- Lysosome related organelles -- Complexity & functions of lysosomal proteins -- Lysosomal storage diseases -- Sphingolipid lysosomal storage diseases as an example -- Lysosomal diseases: multimorbidity disorders -- Pathogenic cascade beyond the Lysosomal System -- Specific therapies approved for some diseases -- Molecular mechanisms and therapeutic approaches of LSDs -- Lysosomal enzymes -- ERTs: approved or in advanced clinical trials -- Approved small molecules: SRT & EET -- Multiple gene therapy trials: enzyme deficiencies -- Cellular organelles targeted by LSD therapies -- Lysosomal & neurodegenerative diseases.…”
Get full text
Series
Electronic Video