Lysosomal storage disorders: Emerging therapeutic options require early diagnosis

European Journal of Pediatrics, Supplement

Volumn 162, Issue 1, 2003, Pages

  Meikle, Peter J ^a   Hopwood, John J ^a  

^a WOMEN S AND CHILDREN S HOSPITAL   (Australia)

Author keywords

Lysosomal storage disorder; Newborn screening; Therapy

Indexed keywords

MERCAPTAMINE; RECOMBINANT ENZYME;

BIOTECHNOLOGY; BLOOD BRAIN BARRIER; BONE MARROW TRANSPLANTATION; CLINICAL TRIAL; CONFERENCE PAPER; CYSTINOSIS; DIAGNOSTIC PROCEDURE; DRUG EFFICACY; DRUG PENETRATION; ENZYME REPLACEMENT; FABRY DISEASE; GAUCHER DISEASE; GLOBOID CELL LEUKODYSTROPHY; GLYCOGEN STORAGE DISEASE TYPE 2; HUMAN; HURLER SYNDROME; LYSOSOME STORAGE DISEASE; METACHROMATIC LEUKODYSTROPHY; MORQUIO SYNDROME; NEWBORN; NEWBORN SCREENING; NONHUMAN; PREVALENCE; PRIORITY JOURNAL; TREATMENT OUTCOME;

EID: 0346059334     PISSN: 09439676     EISSN: None     Source Type: Journal    
DOI: 10.1007/s00431-003-1348-y     Document Type: Conference Paper

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