Treatment of lysosomal storage disorders: Successes and challenges

Journal of Inherited Metabolic Disease

Volumn 37, Issue 4, 2014, Pages 587-598

  Hollak, Carla E M ^a   Wijburg, Frits A ^a  

^a ACADEMIC MEDICAL CENTER   (Netherlands)

Author keywords

[No Author keywords available]

Indexed keywords

CHAPERONE; ANTIOXIDANT;

BIOLOGICAL THERAPY; BLOOD BRAIN BARRIER; CLINICAL EFFECTIVENESS; CLINICAL TRIAL (TOPIC); CONFERENCE PAPER; DISEASE COURSE; ENZYME REPLACEMENT; FABRY DISEASE; GAUCHER DISEASE; GENE THERAPY; GLYCOGEN STORAGE DISEASE TYPE 2; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMAN; LYSOSOME STORAGE DISEASE; MUCOPOLYSACCHARIDOSIS; NONHUMAN; PRACTICE GUIDELINE; SUBSTRATE REDUCTION THERAPY; THERAPY; ANIMAL; LYSOSOMAL STORAGE DISEASES; MOLECULARLY TARGETED THERAPY; PALLIATIVE THERAPY; PROCEDURES;

ANIMALS; ANTIOXIDANTS; ENZYME REPLACEMENT THERAPY; GENETIC THERAPY; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; LYSOSOMAL STORAGE DISEASES; MOLECULAR CHAPERONES; MOLECULAR TARGETED THERAPY; PALLIATIVE CARE;

EID: 84904133859     PISSN: 01418955     EISSN: 15732665     Source Type: Journal    
DOI: 10.1007/s10545-014-9718-3     Document Type: Conference Paper

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