Treatments for lysosomal storage disorders

Biochemical Society Transactions

Volumn 38, Issue 6, 2010, Pages 1465-1468

  Lachmann, Robin ^a  

^a NATIONAL HOSPITAL FOR NEUROLOGY AND NEUROSURGERY   (United Kingdom)

Author keywords

Bone marrow transplantation; Enzyme replacement therapy; Lysosomal storage disorder treatment; Mucopolysaccharidosis; Substrate reduction therapy

Indexed keywords

1 DEOXYGALACTONOJIRIMYCIN; CHAPERONE; GALACTOSE; GENISTEIN; GLUCOSYLCERAMIDASE; IMIGLUCERASE; MIGLUSTAT; PYRIDOXINE; PYRIMETHAMINE; RECOMBINANT ENZYME; RECOMBINANT PROTEIN; SOMATOMEDIN B RECEPTOR; TALIGLUCERASE ALFA; UNCLASSIFIED DRUG; VELAGLUCERASE ALFA;

ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION; BLOOD BRAIN BARRIER; CEREBROSPINAL FLUID; CHO CELL; CLINICAL TRIAL; CONFERENCE PAPER; CRYSTAL STRUCTURE; DRUG EFFICACY; ENZYME ACTIVE SITE; ENZYME ACTIVITY; ENZYME REPLACEMENT; ENZYME SUBSTRATE; FABRY DISEASE; FUCOSIDOSIS; GANGLIOSIDOSIS; GAUCHER DISEASE; GLOBOID CELL LEUKODYSTROPHY; GLYCOGEN STORAGE DISEASE TYPE 2; GLYCOSYLATION; HOMOCYSTINURIA; HUMAN; HUNTER SYNDROME; HURLER SYNDROME; LYSOSOME STORAGE DISEASE; METACHROMATIC LEUKODYSTROPHY; MORQUIO SYNDROME; NIEMANN PICK DISEASE; NONHUMAN; PHENYLKETONURIA; PLACENTA; PRIORITY JOURNAL; SANFILIPPO SYNDROME; THERAPY EFFECT; TREATMENT FAILURE; TREATMENT RESPONSE;

BONE MARROW TRANSPLANTATION; CLINICAL TRIALS AS TOPIC; ENZYME REPLACEMENT THERAPY; GENE THERAPY; HUMANS; LYSOSOMAL STORAGE DISEASES; LYSOSOMES; MOLECULAR CHAPERONES;

EID: 78649742242     PISSN: 03005127     EISSN: 14708752     Source Type: Journal    
DOI: 10.1042/BST0381465     Document Type: Conference Paper

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