Intrathecal enzyme replacement therapy for mucopolysaccharidosis I: Translating success in animal models to patients

Current Pharmaceutical Biotechnology

Volumn 12, Issue 6, 2011, Pages 946-955

  Dickson, Patricia I ^a   Chen, Agnes H ^a  

^a HARBOR UCLA MEDICAL CENTER   (United States)

Author keywords

Enzyme replacement therapy; Hurler; Intrathecal; Lysosomal storage disease; Mucopolysaccharidosis

Indexed keywords

LARONIDASE; RECOMBINANT ENZYME;

ARTICLE; BLOOD BRAIN BARRIER; CEREBROSPINAL FLUID; CEREBROSPINAL FLUID PRESSURE; CLINICAL TRIAL; COGNITION; DIFFUSION TENSOR IMAGING; DISEASE MARKER; ENZYME ACTIVITY; ENZYME DEFICIENCY; ENZYME MODIFICATION; ENZYME REPLACEMENT; EVOKED SOMATOSENSORY RESPONSE; GLOBOID CELL LEUKODYSTROPHY; GM1 GANGLIOSIDOSIS; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HISTOPATHOLOGY; HUMAN; HUNTER SYNDROME; HURLER SYNDROME; INTELLIGENCE QUOTIENT; LAMINECTOMY; LEUKOCYTOSIS; LYSOSOME STORAGE DISEASE; METACHROMATIC LEUKODYSTROPHY; MORQUIO SYNDROME; MUCOPOLYSACCHARIDOSIS TYPE 7; NEUROIMAGING; NEURONAL CEROID LIPOFUSCINOSIS; NEUROPSYCHOLOGICAL TEST; NIEMANN PICK DISEASE; NONHUMAN; NUCLEAR MAGNETIC RESONANCE IMAGING; NUCLEAR MAGNETIC RESONANCE SPECTROSCOPY; OUTCOME ASSESSMENT; RATING SCALE; SANFILIPPO SYNDROME; SCHEIE SYNDROME; SPINAL CORD COMPRESSION; SPINAL CORD DISEASE; TREATMENT OUTCOME;

ANIMALS; CENTRAL NERVOUS SYSTEM DISEASES; CLINICAL TRIALS AS TOPIC; ENZYME REPLACEMENT THERAPY; HUMANS; INJECTIONS, SPINAL; MODELS, ANIMAL; MUCOPOLYSACCHARIDOSIS I;

ANIMALIA;

EID: 79956259719     PISSN: 13892010     EISSN: 18734316     Source Type: Journal    
DOI: 10.2174/138920111795542642     Document Type: Article

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