Emerging therapies and therapeutic concepts for lysosomal storage diseases

Expert Opinion on Orphan Drugs

Volumn 1, Issue 5, 2013, Pages 385-404

  Kirkegaard, Thomas ^a  

^a ORPHAZYME A S   (Denmark)

Author keywords

Chaperone; Enzyme replacement; Fabry disease; Gaucher disease; Glycosphingolipids; Lysosomal storage disease; Lysosomes; Substrate optimization; Substrate reduction; Therapy

Indexed keywords

AGALSIDASE ALFA; AGALSIDASE BETA; CHAPERONE; GALSULFASE; IDURONATE 2 SULFATASE; IMIGLUCERASE; LARONIDASE; LYSOSOME ASSOCIATED MEMBRANE PROTEIN; MERCAPTAMINE; MIGLUSTAT; RECOMBINANT GLUCAN 1,4 ALPHA GLUCOSIDASE; TALIGLUCERASE ALFA; VELAGLUCERASE ALFA;

AUTOPHAGY; BIOSYNTHESIS; BLOOD BRAIN BARRIER; CYSTINOSIS; DRUG DEVELOPMENT; DRUG EFFICACY; DRUG INDUSTRY; DRUG MARKETING; DRUG TOLERABILITY; ENDOCYTOSIS; ENZYME ACTIVITY; ENZYME REPLACEMENT; EXOCYTOSIS; FABRY DISEASE; GAUCHER DISEASE; GENE THERAPY; GENOTYPE PHENOTYPE CORRELATION; GLYCOGEN STORAGE DISEASE TYPE 2; HEALTH CARE NEED; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMAN; HUNTER SYNDROME; HURLER SYNDROME; IMMUNOSUPPRESSIVE TREATMENT; IN VITRO STUDY; IN VIVO STUDY; LYSOSOME; LYSOSOME STORAGE DISEASE; MAROTEAUX LAMY SYNDROME; MONOTHERAPY; MORBIDITY; MORTALITY; NONHUMAN; PATHOPHYSIOLOGY; PHASE 1 CLINICAL TRIAL (TOPIC); PHASE 2 CLINICAL TRIAL (TOPIC); REVIEW; SUBSTRATE REDUCTION THERAPY; THERAPY; TRANS GOLGI NETWORK; TRANSCYTOSIS;

EID: 84896713980     PISSN: None     EISSN: 21678707     Source Type: Journal    
DOI: 10.1517/21678707.2013.780970     Document Type: Review

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