Enzyme replacement in neuronal storage disorders in the pediatric population

Current Treatment Options in Neurology

Volumn 15, Issue 5, 2013, Pages 634-651

  Augustine, Erika F ^a   Mink, Jonathan W ^a  

^a UNIVERSITY OF ROCHESTER MEDICAL CENTER   (United States)

Author keywords

Bone marrow transplant; Enzyme replacement therapy; Fabry disease; Gaucher disease; Glycogen storage disease; Hematopoietic stem cell transplant; Hunter syndrome; Hurler syndrome; Hurler Scheie syndrome; Inborn error of metabolism; Lysosomal storage disease; Mucopolysaccharidoses; Neuronal storage disease; Pompe disease; Scheie syndrome; Treatment

Indexed keywords

AGALSIDASE ALFA; ALGLUCERASE; AMIODARONE; GENTAMICIN; IDURONATE 2 SULFATASE; IMIGLUCERASE; LARONIDASE; PLACEBO; RECOMBINANT GLUCAN 1,4 ALPHA GLUCOSIDASE; VELAGLUCERASE ALFA;

ABDOMINAL PAIN; ANGIONEUROTIC EDEMA; ARTHRALGIA; ARTICLE; CHILD; CLINICAL FEATURE; CLINICAL TRIAL (TOPIC); DIAPHORESIS; DRUG COST; DRUG ERUPTION; ENZYME REPLACEMENT; EXPERIMENTAL ANIMAL; FABRY DISEASE; FACIAL SWELLING; FATIGUE; FEASIBILITY STUDY; FEVER; FLUSHING; GAUCHER DISEASE; GENE THERAPY; GLYCOGEN STORAGE DISEASE TYPE 2; HEADACHE; HUMAN; HUNTER SYNDROME; HURLER SYNDROME; HYPERTENSION; HYPOXEMIA; HYPOXIA; INFUSION RELATED REACTION; INTRATHECAL DRUG ADMINISTRATION; LYSOSOME STORAGE DISEASE; NAUSEA; NEPHROTIC SYNDROME; NEUROLOGIC DISEASE; NEURONAL STORAGE DISORDER; PATIENT CARE; PRURITUS; RHINOPHARYNGITIS; RIGOR; SIDE EFFECT; SUBSTRATE REDUCTION THERAPY; SWELLING; SYMPTOM; TACHYCARDIA; THERAPY; THERAPY EFFECT; URTICARIA; VISUAL DISORDER;

EID: 84885178023     PISSN: 10928480     EISSN: 15343138     Source Type: Journal    
DOI: 10.1007/s11940-013-0256-3     Document Type: Article

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