Current and emerging therapies for the lysosomal storage disorders

Expert Opinion on Emerging Drugs

Volumn 10, Issue 4, 2005, Pages 891-902

  Pastores, Gregory M ^a   Barnett, Natalie L ^a  

^a NEW YORK UNIVERSITY SCHOOL OF MEDICINE   (United States)

Author keywords

Chaperone mediated enzyme enhancement; Enzyme therapy; Gene therapy; Lysosomal storage disorders; Stem cells; Substrate depletion

Indexed keywords

ADENOVIRUS VECTOR; AGALSIDASE ALFA; AGALSIDASE BETA; ALGLUCERASE; ALPHA GALACTOSIDASE; ALPHA GLUCOSIDASE; ANTIHISTAMINIC AGENT; ANTIPYRETIC AGENT; BETA GALACTOSIDASE; BETA GLUCOCEREBROSIDASE; BETA GLUCOSIDASE; BETA N ACETYLHEXOSAMINIDASE; CEREBROSIDE SULFATASE; CHAPERONE; CYSTAMINE; DEOXYGALACTONOJIRIMYCIN; ENZYME; GALACTOSE; GALSULFASE; GLUCOSYLCERAMIDASE; IMIGLUCERASE; LARONIDASE; LENTIVIRUS VECTOR; LYSOSOME ENZYME; MANNOSE 6 PHOSPHATE; MIGLUSTAT; PROPIVERINE; RECOMBINANT ALPHA GLUCOSIDASE; RECOMBINANT ENZYME; RETROVIRUS VECTOR; UNCLASSIFIED DRUG;

ABDOMINAL DISCOMFORT; ASPARTYLGLYCOSAMINURIA; BLOOD BRAIN BARRIER; BLOOD CEREBROSPINAL FLUID BARRIER; BODY WEIGHT DISORDER; BONE MARROW TRANSPLANTATION; CLINICAL TRIAL; COGNITIVE DEFECT; CYSTINOSIS; DIARRHEA; DISEASE COURSE; DRUG EFFICACY; DRUG FEVER; DRUG INDICATION; DRUG SAFETY; ENZYME REPLACEMENT; ETHNOLOGY; FABRY DISEASE; FLATULENCE; GAUCHER DISEASE; GENETIC DISORDER; GENETIC ENGINEERING; GLOBOID CELL LEUKODYSTROPHY; GLYCOGEN STORAGE DISEASE TYPE 2; GRAFT FAILURE; HEALTH CARE COST; HEALTH CARE POLICY; HEMATOPOIETIC STEM CELL; HUMAN; HUNTER SYNDROME; HURLER SYNDROME; LIFESPAN; LYSOSOME STORAGE DISEASE; MAROTEAUX LAMY SYNDROME; MORQUIO SYNDROME; NEUROLOGICAL COMPLICATION; NIEMANN PICK DISEASE; NONHUMAN; NONVIRAL GENE THERAPY; PALLIATIVE THERAPY; PARESTHESIA; PERIPHERAL NEUROPATHY; PREVALENCE; PRURITUS; QUALITY OF LIFE; REVIEW; RIGOR; SANFILIPPO SYNDROME; SIDE EFFECT; STEM CELL TRANSPLANTATION; TAY SACHS DISEASE; TREMOR; URTICARIA; VIRAL GENE THERAPY; WEIGHT REDUCTION;

ANIMALS; BONE MARROW TRANSPLANTATION; DRUGS, INVESTIGATIONAL; GENE THERAPY; HUMANS; LYSOSOMAL STORAGE DISEASES;

EID: 27844491492     PISSN: 14728214     EISSN: None     Source Type: Journal    
DOI: 10.1517/14728214.10.4.891     Document Type: Review

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